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Researchers work to create kidney filtration barrier on a chip Harvard Gazette ... and the stem cell-derived kidney podocytes we developed could even offer a new injectable cell therapy approach for regenerative medicine in patients with life-threatening glomerulopathies in the future, said Ingber, who is director of the Wyss ... and more »

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Researchers work to create kidney filtration barrier on a chip - Harvard Gazette

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The researchers at the University of Pittsburgh have just used the CRISPR-Cas9 genome editing system to forever revolutionize the fight against cancer.

The treatmentwhen used on micewas shown to shrink aggressive tumors and increase survival rates without harming healthy cells. Meaning only cancer cells are attacked, effectively leaving healthy cells unharmed.

CRISPR Cancer Treatment Explained The CRISPR cancer treatment targets fusion genes, which are mutations created when two genes combine to form one hybridoften leading to cancer.

Previously, researchers found MAN2A1-FER, a fusion gene known to be associated with prostate, liver, lung, and ovarian cancer. It also contributes to thegrowth and spread of these tumors.

The unique DNA fingerprint of fusion genes could, however, be its own downfall with the CRISPR cancer treatment targeting specific DNA sequences. The treatment seeks out fusion gene patterns and replaces them with cancer-killing ones.

This is the first time that gene editing has been used to specifically target cancer fusion genes, saidJian-Hua Luo, lead author of the study. Luo added:

The tool lays the groundwork for what could become a totally new approach to treating cancer. Other types of cancer treatments target the foot soldiers of the army. Our approach is to target the command center, so there is no chance for the enemys soldiers to regroup in the battlefield for a comeback.

To test the technique, Luos team transplanted human liver and prostate cancer cells into mice and treated one group with the CRISPR cancer tool to target those fusion genes. The second group was given the same treatment targeting fusion genes that they didnt carry.

From the first group, the mices tumors shrunk up to 30 percent, didnt spread to throughout the body, and all the mice survived the eight-week test.

Meanwhile, the second group had the mices tumors grow nearly 40 times larger, spreading to other parts of the body in most cases. None of the mice in this group made it to the end of the test period.Big Plans For CRISPR-Cas9 The genome editing system has already proven itself to be an incredible tool, giving us new and amazing ways to battle muscular dystrophy, blindness, and HIV.

By also editing human immune cells to more efficiently battle cancer cells, the CRISPR cancer treatment has now been used in human trials.

It truly is an exciting time in the world of medical research as developments continue showing that the CRISPR cancer technique can remit cancer cells.

Despite this, researchers have bigger plans for the CRISPR cancer technique. They plan on testing further in hopes of completely eradicating cancer.

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What You Need to Know About the New CRISPR Cancer Treatment - BOSS Magazine

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Gene therapy can now combine with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 187 companies involved in developing gene therapy are presented along with 232 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.

Key Topics Covered:

Part I: Technologies & Markets

Executive Summary

1. Introduction

2. Gene Therapy Technologies

3. Clinical Applications of Gene Therapy

4. Gene Therapy of Genetic Disorders

5. Gene Therapy of Cancer

6. Gene Therapy of Neurological Disorders

7. Gene Therapy of Cardiovascular Disorders

8. Gene therapy of viral infections

9. Research, Development and Future of Gene Therapy

10. Regulatory, Safety, Ethical Patent Issues of Gene Therapy

11. Markets for Gene Therapy

12. References

Part II: Companies

13. Companies involved in Gene Therapy

For more information about this report visit http://www.researchandmarkets.com/research/9jzl3f/gene_therapy

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-gene-therapy-technologies-markets-and-companies-research-report-2017-2026---research-and-markets-300455149.html

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Global Gene Therapy Technologies, Markets and Companies Research Report 2017-2026 - Research and Markets - PR Newswire (press release)

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"With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program," said Dr. Sandy Macrae, Sangamo's Chief Executive Officer. "We believe Pfizer's end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases."

Under the terms of the collaboration agreement, Sangamo will receive a $70 million upfront payment from Pfizer. Sangamo will be responsible for conducting the SB-525 Phase 1/2 clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products, if any. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.

Gene therapy is a potentially transformational technology for patients, focused on highly specialized, one-time, treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient's cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.

Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A. SB-525 is comprised of a rAAV vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has cleared initiation of human clinical trials for SB-525, which also has been granted orphan drug designation. Sangamo is on track this quarter to start a Phase 1/2 clinical trial to evaluate safety and to measure blood levels of Factor VIII protein and other efficacy endpoints.

Conference CallSangamo will host a conference call today, May 10, 2017 at 5:00 p.m. ET, which will be open to the public, to discuss the details of the collaboration and the Company's first quarter business and financial results. The call will also be webcast live and can be accessed via a link the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A replay of the webcast will also be available for one week after the call.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 15225000. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on May 10, 2017 to 11:59 p.m. ET on May 17, 2017. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 15225000.

About Sangamo Therapeutics Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Pfizer for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.

Forward Looking Statements This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation references relating to the collaboration agreement with Pfizer, potential milestone payments and royalties under the collaboration agreement, ability of the collaboration to advance and commercialize SB-525 as a treatment for Hemophilia A, research and development of therapeutic applications of Sangamo's genomic therapy platforms, the expected timing of clinical trials of lead programs, including SB-525 and the release of data from these trials, the impact of Sangamo's clinical trials on the field of genetic medicine and the benefit of orphan drug status. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to substantial dependence on the clinical success of lead therapeutic programs, the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this press release.

Pfizer and Rare DiseaseRare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide,i representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuroscience, and inherited metabolic disorders.ii

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness and meet the needs of patient families.

Pfizer Inc: Working together for a healthier worldAt Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. For more information, please visit us at http://www.pfizer.com. In addition, to learn more, follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure Notice: The information contained in this release is as of May 10, 2017. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about an investigational Hemophilia A agent, SB-525, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical study commencement and completion dates as well as the possibility of unfavorable study results, including unfavorable new clinical data and additional analyses of existing clinical data; risks associated with initial data, including the risk that the final results of the Phase I/2 study for SB-525 and/or additional clinical trials may be different from (including less favorable than) the initial data results and may not support further clinical development; whether and when any applications may be filed with regulatory authorities for SB-525; whether and when regulatory authorities may approve any such applications, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of SB-525; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

i Rare Disease: Facts and Statistics. http://globalgenes.org/rare-diseases-facts-statistics. Accessed September 7, 2016. ii Pfizer Inc. Rare Disease. http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus. Accessed December 20, 2016.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-and-pfizer-announce-collaboration-for-hemophilia-a-gene-therapy-300455555.html

SOURCE Sangamo Therapeutics, Inc.

http://www.sangamo.com

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Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy - PR Newswire (press release)

Recommendation and review posted by Bethany Smith

Brammer Bio says its commercial-scale gene therapy manufacturing facility in Cambridge, Massachusetts, US, will open in the second half of 2017.

The contract development and manufacturing organization (CDMO) said the site offers process development, clinical phase, and current Good Manufacturing Practices (cGMP) services for cell and gene therapies.

We are delighted to add an experienced commercial biologics team and facilities to help meet the needs of this transformative industry, said Mark Bamforth, CEO of Brammer.

Brammer has completed a Type-C meeting with the US Food and Drug Administration (FDA) to assess the plans for the Massachusetts-based site. A Type-C meeting regards the development and review of drugs or biological drug products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

A Site Master File will be submitted later this year to support Brammers clients product applications.

The site originally housed Biogens clinical and commercial biologics manufacturing facility.

Brammer purchased it along with Biogens distribution center in Somerville, Massachusetts, on January 1, 2017.

The Somerville site offers Brammer nearby storage and distribution capabilities.

The announcement follows Brammers 2016 plans to renovate its 50,000 square-foot facility in Lexington, Massachusetts, to cater for late stage and commercial therapy supply.

Brammers facilities now offer 230,000 square feet of development, distribution and cGMP manufacturing capabilities across Florida and Massachusetts, US.

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Brammer invests in commercial-ready gene therapies - BioPharma-Reporter.com

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The adrenal glands sit atop the kidneys.

The adrenal glands affect metabolism, blood pressure, the immune system, sex hormones and the body's response to stress. The human body has two adrenal glands. About 1 inch by 2 inches (2.54 centimeters by 5.08 cm), they sit on top of the kidneys.

Along with the parathyroid glands, pituitary gland, thyroid gland, pancreas, ovaries (in females) and testicles (in males), the adrenal glands are part of the endocrine system. This system produces hormones that control just about every function in the body.

"The adrenal gland is an intricate part of the HPA (hypothalamus, pituitary, adrenal) Axis," Dr. Mark Engelman, permanent Clinical Consultant for Cyrex Laboratories, told Live Science. "This intimate physiological relationship is fundamental and critical to our wellbeing."

The hypothalamus acts as the body's thermostat, Engelman said. It senses most of the important physiological elements involved in homeostasis and sends out signals to correct perceived unhealthy variations. It connects directly to the pituitary gland, which essentially picks up the orders from the hypothalamus and sends out signals to various organs and glands, including the adrenals, to carry out these orders. The adrenal glands then produce a wide range of hormones, including estrogen, adrenalin and cortisol.

"One of the primary activities of cortisol is to increase available glucose to the nervous system by breaking down protein and fat to glucose in the liver," Engelman said. "It helps block glucose uptake into tissues other than the central nervous system."

Cortisol also has powerful anti-inflammatory and anti-allergy actions, according to Encyclopedia Britannica. It decreases the activities of the immune system to reduce inflammation conditions. Because of this, it is used to treat dermatitis, insect bites, inflammation from arthritis and ulcerative colitis.

One of the most important functions of the adrenal gland is the fight-or-flight response. When a person is stressed or frightened, the adrenal gland releases a flood of hormones, such as adrenaline and cortisol. These hormones increase the heart rate, elevate blood pressure, boost energy supplies, sharpen concentration and slow down other body processes so the body can run from or fight a threat.

Too much of a stress response is a bad thing, though. Too much exposure to elevated hormones from the adrenal gland can cause anxiety, depression, digestive problems, headaches, heart disease, sleep problems, weight gain and memory and concentration impairment, according to the Mayo Clinic.

There are many dysfunctions and diseases associated with the adrenal gland. One of them is the growth of tumors on the glands. These tumors can be benign or cancerous and can throw off the proper production of hormones. A tumor that causes the gland to create too much hormone is called a functioning tumor, while a tumor that causes too little hormone to be produced is called a nonfunctioning tumor. Approximately 4 to 12 out of 1 million people develop a type of adrenal tumor called adrenocortical carcinoma, according to American Society of Clinical Oncology. Some types of adrenal tumors may be linked to ADHD.

A "trendy" disorder is adrenal fatigue. It currently is not an accepted diagnosis option for the medical community at large. The Mayo Clinic defines adrenal fatigue as "a term applied to a collection of nonspecific symptoms, such as body aches, fatigue, nervousness, sleep disturbances and digestive problems." These symptoms are thought to be caused by a wide range of adrenal problems, such as low hormone production.

Engelman said he thinks the signs and symptoms of adrenal fatigue are not related to the ability of the adrenals to work, but rather decreased stimulation from an over-taxed central nervous system. The entire physiological system is based on the concept of maintaining homeostasis. "I have heard from lecturers and read many theories and controversies about 'adrenal fatigue,'" Engelman said. "The ones that make the most scientific sense to me relate to the down regulation of central nervous system receptors to stress signals. This ultimately decreases downstream stimulatory signaling to the adrenal glands as a brain self-protective mechanism from the damaging effects of long term stress."

Adrenal fatigue is thought to be a lesser form of adrenal insufficiency. Adrenal insufficiency (Addison's disease), a condition widely accepted by medical experts. It occurs when the adrenal gland does not produce enough hormones as a result of an underlying disease. Some symptoms are:

Additional resources

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Adrenal Glands: Facts, Function & Disease - Live Science

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ROCKLAND, Mass. and NEW YORK, May 9, 2017 /PRNewswire/ -- EMD Serono, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany, in the US and Canada, and Pfizer Inc. (NYSE: PFE) today announced that the US Food and Drug Administration (FDA) has approved BAVENCIO (avelumab) Injection for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) who have disease progression during or following platinum-containing chemotherapy, or who have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. BAVENCIO was previously granted accelerated approval from the FDA for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC). These indications are approved under accelerated approval based on tumor response and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.1

"This approval for BAVENCIO in patients with locally advanced or metastatic urothelial carcinoma exemplifies our unwavering commitment to finding new treatments for the most challenging cancers," said Luciano Rossetti, M.D., Executive Vice President, Global Head of Research & Development at the biopharma business of Merck KGaA, Darmstadt, Germany. "Coming just a few weeks after the approval for metastatic Merkel cell carcinoma, we continue to demonstrate our ability to accelerate access to innovative medicines for patients in need."

"This approval builds on the ongoing clinical development program for BAVENCIO in urothelial carcinoma and reinforces our commitment to providing new medicines to patients with difficult-to-treat cancers," said Liz Barrett, Global President, Pfizer Oncology. "By drawing on the strength of the alliance, as well as Pfizer's deep experience in genitourinary cancers, we believe BAVENCIO will be an important treatment option, and we hope it will help to improve outcomes for these patients."

Bladder cancer makes up approximately 90% of urothelial carcinomas and is the sixth most common cancer in the US.2,3 When the disease has metastasized, the five-year survival rate is approximately 5%.4 Despite advances in the treatment of locally advanced or metastatic urothelial carcinoma, the prognosis for patients remains poor and more treatment options are needed.2

"Once urothelial carcinoma progresses after treatment with chemotherapy, the five-year survival rate is alarmingly low," said Dr. Andrea Apolo, National Cancer Institute, Bethesda, MD, US. "Until recently, there had been limited innovation in urothelial carcinoma, and this approval gives us another treatment to help battle this aggressive disease."

The efficacy and safety of BAVENCIO was demonstrated in the urothelial carcinoma cohorts (N=242) of the JAVELIN Solid Tumor trial, a Phase I, open-label, single-arm, multicenter study of BAVENCIO in the treatment of various solid tumors. The urothelial carcinoma cohorts enrolled patients with locally advanced or metastatic urothelial carcinoma with disease progression on or after platinum-containing chemotherapy or who had disease progression within 12 months of treatment with a platinum-containing neoadjuvant or adjuvant chemotherapy regimen. These data will be presented at an upcoming medical congress.

The warnings and precautions for BAVENCIO include immune-mediated adverse reactions (such as pneumonitis, hepatitis, colitis, endocrinopathies, nephritis and renal dysfunction and other adverse reactions), infusion-related reactions and embryo-fetal toxicity. The most common adverse reactions (reported in at least 20% of patients) in patients with locally advanced or metastatic urothelial carcinoma were fatigue (41%), infusion-related reaction (30%), musculoskeletal pain (25%), nausea (24%), decreased appetite/hypophagia (21%) and urinary tract infection (21%).1 For more information, please see Important Safety Information for BAVENCIO below.

BAVENCIO is designed to potentially engage both the adaptive and innate immune systems. By binding to PD-L1, BAVENCIO is thought to prevent tumor cells from using PD-L1 for protection against white blood cells, such as T cells, exposing them to anti-tumor responses.1 BAVENCIO has also been shown to induce antibody-dependent cell-mediated cytotoxicity (ADCC) in vitro.1

The alliance is committed to providing industry-leading patient access and reimbursement support through its CoverOne program. This program provides a spectrum of patient access and reimbursement support services intended to help patients receive appropriate access to BAVENCIO in the United States. CoverOne may be reached by phone at 844-8COVER1 (844-826-8371) or online at http://www.CoverOne.com.

About Urothelial Carcinoma Cohorts in JAVELIN Solid Tumor Trial The efficacy and safety of BAVENCIO was demonstrated in the urothelial carcinoma cohorts of the JAVELIN Solid Tumor trial, a Phase I, open-label, single-arm, multicenter study that included 242 patients with locally advanced or metastatic urothelial carcinoma with disease progression on or after platinum- containing chemotherapy or who had disease progression within 12 months of treatment with a platinum-containing neoadjuvant or adjuvant chemotherapy regimen who were treated with BAVENCIO.

Patients with active or a history of central nervous system metastasis; other malignancies within the last five years; an organ transplant; conditions requiring therapeutic immune suppression; or active infection with HIV, hepatitis B or C were excluded. Patients with autoimmune disease, other than type 1 diabetes, vitiligo, psoriasis, or thyroid disease that did not require immunosuppressive treatment, were excluded. Patients were included regardless of their PD-L1 status. Patients received BAVENCIO at a dose of 10 mg/kg intravenously over 60 minutes every two weeks until disease progression or unacceptable toxicity. Tumor response assessments were performed every six weeks, as assessed by an Independent Endpoint Review Committee (IERC) using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Efficacy outcome measures included confirmed overall response rate, (ORR) and duration of response (DOR). Efficacy measures were evaluated in patients who were followed for a minimum of both 13 weeks and 6 months at the time of data cut-off.

Out of the total 226 patients evaluable for efficacy, 44% had non-bladder urothelial carcinoma, including 23% of patients with upper tract disease; 83% of patients had visceral metastases; 34% of patients had liver metastases. Nine patients (4%) had disease progression following prior platinum-containing neoadjuvant or adjuvant therapy only. Forty-seven percent of patients only received prior cisplatin-based regimens, 32% received only prior carboplatin-based regimens, and 20% received both cisplatin and carboplatin-based regimens.

The international clinical development program for avelumab, known as JAVELIN, involves more than 30 clinical programs, including nine Phase III trials, and more than 5,200 patients across more than 15 tumor types.

In December 2015, Merck KGaA, Darmstadt, Germany and Pfizer announced the initiation of a Phase III multicenter, multinational, randomized, open-label, parallel-arm study (JAVELIN Bladder 100) of BAVENCIO plus best supportive care versus best supportive care alone as a maintenance treatment in patients with locally advanced or metastatic urothelial carcinoma whose disease did not progress after completion of first-line platinum-containing chemotherapy. This trial is currently enrolling patients.

For more information about JAVELIN trials, please visit http://www.clinicaltrials.gov.

For full prescribing information and medication guide for BAVENCIO, please see http://www.BAVENCIO.com.

IMPORTANT SAFETY INFORMATION and INDICATIONS

BAVENCIO can cause immune-mediated pneumonitis, including fatal cases. Monitor patients for signs and symptoms of pneumonitis and evaluate suspected cases with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold BAVENCIO for moderate (Grade 2) and permanently discontinue for severe (Grade 3), life-threatening (Grade 4), or recurrent moderate (Grade 2) pneumonitis. Pneumonitis occurred in 1.2% (21/1738) of patients, including one (0.1%) patient with Grade 5, one (0.1%) with Grade 4, and five (0.3%) with Grade 3.

BAVENCIO can cause immune-mediated hepatitis, including fatal cases. Monitor patients for abnormal liver tests prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater hepatitis. Withhold BAVENCIO for moderate (Grade 2) immune-mediated hepatitis until resolution and permanently discontinue for severe (Grade 3) or life-threatening (Grade 4) immune-mediated hepatitis. Immune-mediated hepatitis was reported in 0.9% (16/1738) of patients, including two (0.1%) patients with Grade 5 and 11 (0.6%) with Grade 3.

BAVENCIO can cause immune-mediated colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold BAVENCIO until resolution for moderate or severe (Grade 2 or 3) colitis and permanently discontinue for life-threatening (Grade 4) or recurrent (Grade 3) colitis upon re-initiation of BAVENCIO. Immune-mediated colitis occurred in 1.5% (26/1738) of patients, including seven (0.4%) with Grade 3.

BAVENCIO can cause immune-mediated endocrinopathies, including adrenal insufficiency, thyroid disorders, and type 1 diabetes mellitus.

Monitor patients for signs and symptoms of adrenal insufficiency during and after treatment, and administer corticosteroids as appropriate. Withhold BAVENCIO for severe (Grade 3) or life-threatening (Grade 4) adrenal insufficiency. Adrenal insufficiency was reported in 0.5% (8/1738) of patients, including one (0.1%) with Grade 3.

Thyroid disorders can occur at any time during treatment. Monitor patients for changes in thyroid function at the start of treatment, periodically during treatment, and as indicated based on clinical evaluation. Manage hypothyroidism with hormone replacement therapy and hyperthyroidism with medical management. Withhold BAVENCIO for severe (Grade 3) or life- threatening (Grade 4) thyroid disorders. Thyroid disorders including hypothyroidism, hyperthyroidism, and thyroiditis were reported in 6% (98/1738) of patients, including three (0.2%) with Grade 3.

Type 1 diabetes mellitus including diabetic ketoacidosis: Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Withhold BAVENCIO and administer anti-hyperglycemics or insulin in patients with severe or life-threatening (Grade 3) hyperglycemia, and resume treatment when metabolic control is achieved. Type 1 diabetes mellitus without an alternative etiology occurred in 0.1% (2/1738) of patients, including two cases of Grade 3 hyperglycemia.

BAVENCIO can cause immune-mediated nephritis and renal dysfunction. Monitor patients for elevated serum creatinine prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater nephritis. Withhold BAVENCIO for moderate (Grade 2) or severe (Grade 3) nephritis until resolution to Grade 1 or lower. Permanently discontinue BAVENCIO for life-threatening (Grade 4) nephritis. Immune-mediated nephritis occurred in 0.1% (1/1738) of patients.

BAVENCIO can result in other severe and fatal immune-mediated adverse reactions involving any organ system during treatment or after treatment discontinuation. For suspected immune-mediated adverse reactions, evaluate to confirm or rule out an immune-mediated adverse reaction and to exclude other causes. Depending on the severity of the adverse reaction, withhold or permanently discontinue BAVENCIO, administer high-dose corticosteroids, and initiate hormone replacement therapy if appropriate. Resume BAVENCIO when the immune-mediated adverse reaction remains at Grade 1 or lower following a corticosteroid taper. Permanently discontinue BAVENCIO for any severe (Grade 3) immune-mediated adverse reaction that recurs and for any life-threatening (Grade 4) immune-mediated adverse reaction. The following clinically significant immune-mediated adverse reactions occurred in less than 1% of 1738 patients treated with BAVENCIO: myocarditis with fatal cases, myositis, psoriasis, arthritis, exfoliative dermatitis, erythema multiforme, pemphigoid, hypopituitarism, uveitis, Guillain-Barr syndrome, and systemic inflammatory response.

BAVENCIO can cause severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions. Patients should be premedicated with an antihistamine and acetaminophen prior to the first 4 infusions and for subsequent doses based upon clinical judgment and presence/severity of prior infusion reactions. Monitor patients for signs and symptoms of infusion-related reactions, including pyrexia, chills, flushing, hypotension, dyspnea, wheezing, back pain, abdominal pain, and urticaria. Interrupt or slow the rate of infusion for mild (Grade 1) or moderate (Grade 2) infusion-related reactions. Permanently discontinue BAVENCIO for severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions. Infusion-related reactions occurred in 25% (439/1738) of patients, including three (0.2%) patients with Grade 4 and nine (0.5%) with Grade 3.

BAVENCIO can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to a fetus including the risk of fetal death. Advise females of childbearing potential to use effective contraception during treatment with BAVENCIO and for at least 1 month after the last dose of BAVENCIO. It is not known whether BAVENCIO is excreted in human milk. Advise a lactating woman not to breastfeed during treatment and for at least 1 month after the last dose of BAVENCIO due to the potential for serious adverse reactions in breastfed infants.

The most common adverse reactions (all grades, 20%) in patients with metastatic Merkel cell carcinoma (MCC) were fatigue (50%), musculoskeletal pain (32%), diarrhea (23%), nausea (22%), infusion-related reaction (22%), rash (22%), decreased appetite (20%), and peripheral edema (20%).

Selected treatment-emergent laboratory abnormalities (all grades, 20%) in patients with metastatic MCC were lymphopenia (49%), anemia (35%), increased aspartate aminotransferase (34%), thrombocytopenia (27%), and increased alanine aminotransferase (20%).

The most common adverse reactions (all grades, 20%) in patients with locally advanced or metastatic urothelial cancer (UC) were fatigue (41%), infusion-related reaction (30%), musculoskeletal pain (25%), nausea (24%), decreased appetite/hypophagia (21%) and urinary tract infection (21%).

Selected laboratory abnormalities (grades 3-4, 3%) in patients with locally advanced or metastatic UC were hyponatremia (16%), gamma-glutamyltransferase increased (12%), lymphopenia (11%), hyperglycemia (9%), increased alkaline phosphatase (7%), anemia (6%), increased lipase (6%), hyperkalemia (3%), and increased aspartate aminotransferase (3%).

INDICATIONS

BAVENCIO is indicated for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC).

BAVENCIO is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

These indications are approved under accelerated approval based on tumor response and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

Please see full Prescribing Information and Medication Guide.

Avelumab has not yet been approved for any indication in any market outside of the US. As announced on October 31, 2016, the European Medicines Agency (EMA) has validated for review Merck KGaA, Darmstadt, Germany's Marketing Authorization Application for avelumab, for the proposed indication of metastatic Merkel cell carcinoma.

About BAVENCIO (avelumab) BAVENCIO is a human programmed death ligand-1 (PD-L1) blocking antibody indicated in the US for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or who have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, as well as for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma.1 These indications are approved under accelerated approval based on tumor response and duration of response. Continued approval for these indications is contingent upon verification and description of clinical benefit in confirmatory trials.

BAVENCIO is not approved for any indication in any market outside the US.

Alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc., New York, US Immuno-oncology is a top priority for Merck KGaA, Darmstadt, Germany and Pfizer Inc. The global strategic alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc., New York, US, enables the companies to benefit from each other's strengths and capabilities and further explore the therapeutic potential of avelumab, an anti-PD-L1 antibody initially discovered and developed by Merck KGaA, Darmstadt, Germany. The immuno-oncology alliance will jointly develop and commercialize avelumab and advance Pfizer's PD-1 antibody. The alliance is focused on developing high-priority international clinical programs to investigate avelumab as a monotherapy, as well as in combination regimens, and is striving to find new ways to treat cancer.

About EMD Serono, Inc. EMD Serono is the biopharmaceutical business of Merck KGaA, Darmstadt, Germany a leading science and technology company in the US and Canada focused exclusively on specialty care. For more than 40 years, the business has integrated cutting-edge science, innovative products and industry-leading patient support and access programs. EMD Serono has deep expertise in neurology, fertility and endocrinology, as well as a robust pipeline of potential therapies in oncology, immuno-oncology and immunology as R&D focus areas. Today, the business has 1,200 employees around the country with commercial, clinical and research operations based in the company's home state of Massachusetts. http://www.emdserono.com

About Merck KGaA, Darmstadt, Germany All Merck KGaA, Darmstadt, Germany Press Releases are distributed by e-mail at the same time they become available on the Merck KGaA, Darmstadt, Germany Website. Please go to http://www.emdgroup.com/subscribe to register online, change your selection or discontinue this service.

Merck KGaA, Darmstadt, Germany, is a leading science and technology company in healthcare, life science and performance materials. Around 50,000 employees work to further develop technologies that improve and enhance life from biopharmaceutical therapies to treat cancer or multiple sclerosis, cutting-edge systems for scientific research and production, to liquid crystals for smartphones and LCD televisions. In 2016, Merck KGaA, Darmstadt, Germany, generated sales of 15.0 billion in 66 countries.

Founded in 1668, Merck KGaA, Darmstadt, Germany, is the world's oldest pharmaceutical and chemical company. The founding family remains the majority owner of the publicly listed corporate group. Merck KGaA, Darmstadt, Germany, holds the global rights to the "Merck" name and brand except in the United States and Canada, where the company operates as EMD Serono, MilliporeSigma and EMD Performance Materials.

Pfizer Inc.: Working together for a healthier world At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @PfizerNews, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure Notice The information contained in this release is as of May 9, 2017. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about BAVENCIO (avelumab), the alliance between Merck KGaA, Darmstadt, Germany and Pfizer involving anti-PD-L1 and anti-PD-1 therapies, and clinical development plans, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of BAVENCIO; the uncertainties inherent in research and development, including the ability to meet anticipated clinical study commencement and completion dates and regulatory submission dates, as well as the possibility of unfavorable study results, including unfavorable new clinical data and additional analyses of existing clinical data; risks associated with interim data; the risk that clinical trial data are subject to differing interpretations, and, even when we view data as sufficient to support the safety and/or effectiveness of a product candidate, regulatory authorities may not share our views and may require additional data or may deny approval altogether; whether and when drug applications may be filed in any other jurisdictions for the Indication or in any jurisdictions for any other potential indications for BAVENCIO, combination therapies or other product candidates; whether and when any such applications (including the pending application for BAVENCIO for metastatic Merkel cell carcinoma in the EU) may be approved by regulatory authorities, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of BAVENCIO, combination therapies or other product candidates; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2016, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

References

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AYTU BIOSCIENCE, INC. (OTCMKTS:AYTU) Files An 8-K Entry into a Material Definitive Agreement Market Exclusive Aytu BioScience, Inc. is a commercial-stage healthcare company focused on acquiring, developing and commercializing products in the field of urology. The Company focuses on hypogonadism, prostate cancer, urinary tract infections and male infertility. Aytu BioScience Announces the Acquisition of Nuelle, Inc., Developer and Marketer of Fiera Female Personal Care ...PR Newswire (press release) all 3 news articles »

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Stems Cells Could Help Treat Slipped Discs Technology Networks The study on the sick German shepherds was organized as follows: With the permission of the dog owners, neurologist Frank Steffen and his team removed stem cells from the marrow of the pelvic bone of the affected animals. After the cleaning and ... and more »

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Iran is expanding investment in stem cell research and its application in various therapies, particularly for hard-to-treat diseases, through the Office of the Vice- Presidency for Science and Technology. As a result of the increase in the number of companies active in the domain of stem cells in the past three years, more than 400 products are processed in the country, indicating a multifold growth compared with the eight-year tenure of previous administration when there were fewer than 50 knowledge-based firms in total.

There are over 40 knowledgebased firms in the field of stem cell and regenerative medicine alone in Iran today, said Amir Ali Hamidiyeh, secretary of the Headquarters for Development of Stem Cell Science and Technology (HDSCST). He made the statement at a press briefing for the second National Festival and International Congress on Stem Cell Sciences and Technologies and Regenerative Medicine to be held July 13- 15 in Tehran, Mehr News Agency reports. According to the conference secretariat, 1,444 people have signed up to attend the event from across the world, including from Iraq, India, Pakistan, Jordan, Russia, Australia, Germany, China, Britain and South Korea. They all are among their countrys respected figures in centers with high academic standing.

The congress is co-sponsored by the Vice-Presidency for Science and Technology and Council on Development of Stem Cell Sciences and Technology. So far, eight stem-cell therapy products for use in hospitals have been produced at the HDSCST laboratories. Manufacturinglicenses have been granted for anadditional number, while others are on thewait list.

Prior to 2014, only 25 knowledgebased companies had applied to operate in this field, of which only one was actively producing quality stem cell products, Hamidiyeh pointed out.

But since then, over 25 workgroups have been formed in cooperation with experts in the specific sciences. Stem cells are cells that have the ability to divide and develop into many different cell types in the body during early life and growth. Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

Future of Medicine in Stem

Cells The future of medicine is interrelated with stem cell therapy and the treatment ofrefractory and incurable diseases is in this field of medicine, according to Dr. Ahmad Vosouq Dizaj, the clinical deputy of Royan Institute. Having access to engineering sciences as well as the combination of biology and medicine can play a crucial role in redressing health problems, he said. Stem cells have the ability to replace damaged cells and treat disease. They can also be used to study diseases and provide a resource for testing new medical treatments. The use of stem cells reduces the risk of viral diseases transmission and incidence of Graft Versus Host Disease (GVHD). The ability to perform organ transplants is among the benefits ofumbilical cord blood transfusion.Using stems cells is also one of thebest ways to treat blood diseases sincethe method has a success rate of 70%worldwide.

Storage of stem cells is a valuable investment. So far, 27 cord blood banks have been launched across the country. There are two types: public and private banks for stem cell storage. The former does not charge a fee for storage. But in the latter, the cost of collection and genetictesting is about $645 and the annualcharge for storage is $33, according toISNA.Iran is a leading country in biomedicalresearch. Researchers and physicians have been successfully performing bone marrow transplants during the past fewyears.Irans stem cell research is centeredat the Royan Institute for ReproductiveBiomedicine, Stem Cell Biology andTechnology, located in northern Tehran.

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Get burned over the weekend? RenovaCare has got your back. The New York-based biotech company has expertise in stem cells and organ regeneration, and has brought these skills to bear on wound care. One of the companys most promising methods uses a literal skin gun to spray skin stem cells on a burn or chronic wound to promote rapid healing. The healing is so rapid that you can walk into the hospital with a burn on a Friday night and return on Monday largelyhealed.

The skin gun process uses a patients stem cells, which are collected from healthy skin. The stem cells are isolated from the skin sample and suspended in a water solution that makes them easy to spray. Thecomputer-controlled skin gun works like the air brushes that are used by painters, but with much more precision.

The treatment is stupidly simple just spray the stem cells on the burned skin and wait for them to regrow. It is also extremely fast, taking only 1.5 hours to isolate the cells and and spray the skin. Once the skin cells are applied, it takes only a few days for the treatment to be effective. When state trooper Matthew Uram was burned in an unfortunate bonfire accident, he chose this experimental treatment and was entirely healed from his second-degree burns in four days.

This skin gun approach offers a significant improvement over the current methods of in-lab skin growth and surgical grafting that takes weeks and sometimes even months to be effective. Those who undergo these conventional skin graft techniques often suffer from infections and other setbacks, rendering the treatment far from optimal. A technology like the skin gun that could promote complete healing in a matter of days would represent a clear advance.

RenovaCares skin gun is still in the developmental stage and has not been approved by the FDA for sale in the United States, so you wont be able to find it on the shelves of burn units quite yet. The company is making progress towards that goal, however, and has recently announceda successful round of testing that shows its gun is capable of dispersing the skin cell liquid in a very uniform and dense manner.

Recent experiments conducted at Stem Cell Systems GmbH (Berlin, Germany) show that the gun can spray more than 20,000 evenly distributed droplets in a test area as compared to a conventional needle and syringe which produced only 91. The gun is not only capable of even dispersal, but it also is gentle on the skin stem cells, which retain 97.3 percent viability after SkinGun spraying. RenovaCare is continuing its research and development as it moves towards FDA approval and eventual commercial rollout. The company recently a filed a 510(k) submission with the FDA, which is a notice of intent to market a device and often is the first step before clinical trials.

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A molecular switch has been identified that converts skin cells into cells found in blood vessels, raising hopes of aiding heart disease sufferers.

This technique boosts levels of an enzyme that keeps cells young and could also potentially help cells avoid ageing as they are grown in the lab. Although this technique has been used before, this is the first time it has been understood by scientists.

Some techniques to convert mature skin cells into pluripotent stem cells use a cocktail of chemicals to ensure they turn into designated cell types. Other methods modify cells, skippingthe stem cell state completely. Recently, researchers have been exploring rewinding skin cells so they lose some of their mature cell identity.

Dr Jalees Rehman, who led the study at the University of Illinois at Chicago, said: They dont revert all the way back to a pluripotent stem cell, but instead turn into intermediate progenitor cells. Even though they only differentiate into a few different cell types, progenitor cells can be grown in large quantities, making them suitable for regenerative therapies.

Rehmans research group discovered that progenitor cells could be converted into blood vessel endothelial cells or erythrocytes depending on the level of a gene transcription factor called SOX17. When SOX17 levels were increased, progenitor cells were five times as likely to become endothelial cells. When this process was reversed, fewer endothelial cells but more erythrocytes were produced.

Dr Rehman said: It makes a lot of sense that SOX17 is involved because it is abundant in developing embryos when blood vessels are forming. When human progenitor cells were embedded into a gel implanted into mice, the cells formed functional human blood vessels. Mice that were suffering from heart damage formed functional human blood vessels in their hearts even interlinking with existing murine vessels to improve heart function.

During the course of the research, the scientists observed increased levels of telomerase the anti-ageing enzyme responsible for telomeres on the ends of chromosome in progenitor cells. The increase in telomerase we see in the progenitor cells could be an added benefit of using this partial de-differentiation technique for the production of new blood vessels for patients with cardiac disease, especially for older patients, said Dr Rehman. The process of converting and expanding these cells in the lab could make them age even further and impair their long-term function. But if the cells have elevated levels of telomerase, the cells are at lower risk of premature ageing.

Increased levels of telomerase are also observed in cancer cells, enabling cell division to occur at avery high rate. However, the scientists didnt observe any tumour formation during their research and their next steps will involve further research over a longer time period in larger animals. The study was published in Circulation.

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Protein enables scientists to convert skin to blood vessels - Lab News

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Scientists may have found a cure for gray hair and baldness by accident Island Packet A protein commonly associated with nerve development, KROX20, turns on in skin cells that turn into a hair shaft. These cells then produce a protein called stem cell factor, which researchers said was key to pigmentation in hair. ... Le said the team's ... and more »

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Scientists may have found a cure for gray hair and baldness by accident - Island Packet

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Shinya Yamanaka ( , Yamanaka Shin'ya?, born September 4, 1962) is a Japanese Nobel Prize-winning stem cell researcher.[1][2][3] He serves as the director of Center for iPS Cell (induced Pluripotent Stem Cell) Research and Application and a professor at the Institute for Frontier Medical Sciences(ja) at Kyoto University; as a senior investigator at the UCSF-affiliated J. David Gladstone Institutes in San Francisco, California; and as a professor of anatomy at University of California, San Francisco (UCSF). Yamanaka is also a past president of the International Society for Stem Cell Research (ISSCR).

He received the 2010 BBVA Foundation Frontiers of Knowledge Award in Biomedicine category. Also he received the Wolf Prize in Medicine in 2011 with Rudolf Jaenisch;[6] the Millennium Technology Prize in 2012 together with Linus Torvalds. In 2012 he and John Gurdon were awarded the Nobel Prize for Physiology or Medicine for the discovery that mature cells can be converted to stem cells.[7] In 2013 he was awarded the $3 million Breakthrough Prize in Life Sciences for his work.

Yamanaka was born in Higashisaka Japan in 1962. After graduating from Tennji High School attached to Osaka Kyoiku University,[8] he received his M.D. at Kobe University in 1987 and his PhD at Osaka City University Graduate School in 1993. After this, he went through a residency in orthopedic surgery at National Osaka Hospital and a postdoctoral fellowship at the Gladstone Institute of Cardiovascular Disease, San Francisco.

Afterwards he worked at the Gladstone Institutes in San Francisco, USA and Nara Institute of Science and Technology in Japan. Yamanaka is currently a Professor at Kyoto University, where he directs its Center for iPS Research and Application. He is also a senior investigator at the Gladstone Institutes as well as the director of the Center for iPS Cell Research and Application(ja).[9]

Between 1987 and 1989, Yamanaka was a resident in orthopedic surgery at the National Osaka Hospital. His first operation was to remove a benign tumor from his friend Shuichi Hirata, a task he could not complete after one hour when a skilled surgeon would have taken ten minutes or so. Some seniors referred to him as "Jamanaka", a pun on the Japanese word for obstacle.[10]

From 1993 to 1996, he was at the Gladstone Institute of Cardiovascular Disease. Between 1996 and 1999, he was an assistant professor at Osaka City University Medical School, but found himself mostly looking after mice in the laboratory, not doing actual research.[10]

His wife advised him to become a practicing doctor, but instead he applied for a position at the Nara Institute of Science and Technology. He stated that he could and would clarify the characteristics of embryonic stem cells, and this can-do attitude won him the job. From 19992003, he was an associate professor there, and started the research that would later win him the 2012 Nobel Prize. He became a full professor and remained at the institute in that position from 20032005. Between 2004 and 2010, Yamanaka was a professor at the Institute for Frontier Medical Sciences.[11] Currently, Yamanaka is the director and a professor at the Center for iPS Cell Research and Application at Kyoto University.

In 2006, he and his team generated induced pluripotent stem cells (iPS cells) from adult mouse fibroblasts.[1] iPS cells closely resemble embryonic stem cells, the in vitro equivalent of the part of the blastocyst (the embryo a few days after fertilization) which grows to become the embryo proper. They could show that his iPS cells were pluripotent, i.e. capable of generating all cell lineages of the body. Later he and his team generated iPS cells from human adult fibroblasts,[2] again as the first group to do so. A key difference from previous attempts by the field was his team's use of multiple transcription factors, instead of transfecting one transcription factor per experiment. They started with 24 transcription factors known to be important in the early embryo, but could in the end reduce it to 4 transcription factors Sox2, Oct4, Klf4 and c-Myc.[1]

Yamanaka practiced judo (2nd Dan black belt) and played rugby as a university student. He also has a history of running marathons. After a 20-year gap, he competed in the inaugural Osaka Marathon in 2011 as a charity runner with a time of 4:29:53. He also took part in the 2012 Kyoto Marathon to raise money for iPS research, finishing in 4:03:19. He also ran in the second Osaka Marathon on November 25, 2012.[12]

In 2007, Yamanaka was recognized as a "Person Who Mattered" in the Time Person of the Year edition of Time Magazine.[13] Yamanaka was also nominated as a 2008 Time 100 Finalist.[14] In June 2010, Yamanaka was awarded the Kyoto Prize for reprogramming adult skin cells to pluripotential precursors. Yamanaka developed the method as an alternative to embryonic stem cells, thus circumventing an approach in which embryos would be destroyed.

In May 2010, Yamanaka was given "Doctor of Science honorary degree" by Mount Sinai School of Medicine.[15]

In September 2010, he was awarded the Balzan Prize for his work on biology and stem cells.[16]

Yamanaka has been listed as one of the 15 Asian Scientists To Watch by Asian Scientist magazine on May 15, 2011.[17][18] In June 2011, he was awarded the inaugural McEwen Award for Innovation; he shared the $100,000 prize with Kazutoshi Takahashi(ja), who was the lead author on the paper describing the generation of induced pluripotent stem cells.[19]

In June 2012, he was awarded the Millennium Technology Prize for his work in stem cells.[20] He shared the 1.2 million euro prize with Linus Torvalds, the creator of the Linux kernel.

In October 2012, he and fellow stem cell researcher John Gurdon were awarded the Nobel Prize in Physiology or Medicine "for the discovery that mature cells can be reprogrammed to become pluripotent."[21]

The 2012 Nobel Prize in Physiology or Medicine was awarded jointly to Sir John B. Gurdon and Shinya Yamanaka "for the discovery that mature cells can be reprogrammed to become pluripotent."[22]

There are different types of stem cells

. These are some types of cells that will help in understanding the material.

totipotency remains through the first few cell divisions ex. the fertilised egg

The early embryo consists mainly of pluripotent stem cells

ex) blood multipotent cells can develop into various blood cells

Theoretically patient-specific transplantations possible

Much research done Immune rejection reducible via stem cell bank

Pluripotent

Abnormal aging

No immune rejection Safe (clinical trials)

The prevalent view during the early 20th century was that mature cells were permanently locked into the differentiated state and cannot return to a fully immature, pluripotent stem cell state. They thought that cellular differentiation can only be a unidirectional process. Therefore, non-differentiated egg/early embryo cells can only develop into specialized cells. However, stem cells with limited potency (adult stem cells) remain in bone marrow, intestine, skin etc. to act as a source of cell replacement.[23]

The fact that differentiated cell types had specific patterns of proteins suggested irreversible epigenetic modifications or genetic alterations to be the cause of unidirectional cell differentiation. So, cells progressively become more restricted in the differentiation potential and eventually lose pluripotency.[24]

In 1962, John B. Gurdon demonstrated that the nucleus from a differentiated frog intestinal epithelial cell can generate a fully functional tadpole via transplantation to an enucleated egg. Gurdon used somatic cell nuclear transfer (SCNT) as a method to understand reprogramming and how cells change in specialization. He concluded that differentiated somatic cell nuclei had the potential to revert to pluripotency. This was a paradigm shift during the time. It showed that a differentiated cell nucleus has retained the capacity to successfully revert to an undifferentiated state, with the potential to restart development (pluripotent capacity).

However, the question still remained whether an intact differentiated cell could be fully reprogrammed to become pluripotent.

Shinya Yamanaka proved that introduction of a small set of transcription factors into a differentiated cell was sufficient to revert the cell to a pluripotent state. Yamanaka focused on factors that are important for maintaining pluripotency in embryonic stem (ES) cells. Knowing that transcription factors were involved in the maintenance of the pluripotent state, he selected a set of 24 ES cell transcriptional factors as candidates to reinstate pluripotency in somatic cells.

First, he collected the 24 candidate factors. When all 24 genes encoding these transcription factors were introduced into skin fibroblasts, few actually generated colonies that were remarkably similar to ES cells. Secondly, further experiments were conducted with smaller numbers of transcription factors added to identify the key factors, through a very simple and yet sensitive assay system. Lastly, he identified the four key factors. They found that 4 transcriptional factors (Myc, Oct3/4, Sox2 and Klf4) were sufficient to convert mouse embryonic or adult fibroblasts to pluripotent stem cells (capable of producing teratomas in vivo and contributing to chimeric mice).

These pluripotent cells are called iPS (induced pluripotent stem) cells; they appeared with very low frequency.

iPS cells can be selected by inserting the b-geo gene into the Fbx15 locus. The Fbx15 promoter is active in pluripotent stem cells which induce b-geo expression, which in turn gives rise to G418 resistance; this resistance helps us identify the iPS cells in a culture.

Moreover, in 2007, Yamanaka and his colleagues found iPS cells with germ line transmission (via selecting for Oct4 or Nanog gene). Also in 2007, they were the first to produce human iPS cells.

However, there are some difficulties to overcome. The first is the issue of the very low production rate of iPS cells, and the other is the fact that the 4 transcriptional factors are shown to be oncogenic.

Nonetheless, this is a truly fundamental discovery. This was the first time an intact differentiated somatic cell could be reprogrammed to become pluripotent. This opened up a completely new research field.

In July 2014, a scandal regarding the research of Haruko Obokata was connected to Yamanaka. He could not find the lab notes from the period in question [25] and was made to apologise.[26][27]

Since the original discovery by Yamanaka, much further research has been done in this field, and many improvements have been made to the technology. Here we[who?] discuss the improvements made to Yamanaka's research as well as the future prospects of his findings.

1. The delivery mechanism of pluripotency factors has been improved. At first retroviral vectors, that integrate randomly in the genome and cause deregulation of genes that contribute to tumor formation, were used. However, now, non-integrating viruses, stabilised RNAs or proteins, or episomal plasmids (integration-free delivery mechanism) are used.

2. Transcription factors required for inducing pluripotency in different cell types have been identified (e.g. neural stem cells).

3. Small substitutive molecules were identified, that can substitute for the function of the transcription factors.

4. Transdifferentiation experiments were carried out. They tried to change the cell fate without proceeding through a pluripotent state. They were able to systematically identify genes that carry out transdifferentiation using combinations of transcription factors that induce cell fate switches. They found trandifferentiation within germ layer and between germ layers, e.g., exocrine cells to endocrine cells, fibroblast cells to myoblast cells, fibroblast cells to cardiomyocyte cells, fibroblast cells to neurons

5. Cell replacement therapy with iPS cells is a possibility. Stem cells can replace diseased or lost cells in degenerative disorders and they are less prone to immune rejection. However, there is a danger that it may introduce mutations or other genomic abnormalities that render it unsuitable for cell therapy. So, there are still many challenges, but it is a very exciting and promising research area. Further work is required to guarantee safety for patients.

6. Can medically use iPS cells from patients with genetic and other disorders to gain insights into the disease process. - Amyotrophic lateral sclerosis (ALS), Rett syndrome, spinal muscular atrophy (SMA), 1-antitrypsin deficiency, familial hypercholesterolemia and glycogen storage disease type 1A. - For cardiovascular disease, Timothy syndrome, LEOPARD syndrome, type 1 and 2 long QT syndrome - Alzheimers, Spinocerebellar ataxia, Huntingtons etc.

7. iPS cells provide screening platforms for development and validation of therapeutic compounds. For example, kinetin was a novel compound found in iPS cells from familial dysautonomia and beta blockers & ion channel blockers for long QT syndrome were identified with iPS cells.

Yamanaka's research has opened a new door and the world's scientists have set forth on a long journey of exploration, hoping to find our cells true potential.[28]

In 2013, iPS cells were used to generate a human vascularized and functional liver in mice in Japan. Multiple stem cells were used to differentiate the component parts of the liver, which then self-organized into the complex structure. When placed into a mouse host, the liver vessels connected to the hosts vessels and performed normal liver functions, including breaking down of drugs and liver secretions. [29]

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Shinya Yamanaka - Wikipedia

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The kidney is made up of about a million tiny units that filter blood, rid the body of undesired waste products while holding back blood cells and valuable proteins, and control the bodys fluid content. Key to each of these units is a structure known the glomerulus, in which podocyte cells wrap themselves tightly around a tuft of capillaries separated only by a thin membrane composed of extracellular matrix, and leave slits between them to build an actual filtration barrier. Podocytes are the target of many congenital or acquired kidney diseases, and they are often harmed by drugs.

To build an in vitro model of the human glomerulus to probe deeper into its function and vulnerabilities to disease and drug toxicities, researchers have been attempting to engineer human stem cells that in theory can give rise to any mature cell type so that they form into functional podocytes. These cell culture efforts, however, so far have failed to produce populations of mature podocytes pure enough as to be useful for modeling glomerular filtration.

A team led by Donald Ingber, Professor of Bioengineering at the Harvard John A. Paulson School of Engineering and Applied Sciences (SEAS) and Founding Director of Harvards Wyss Institute of Biologically Inspired Engineering, now reports a solution to this challenge in Nature Biomedical Engineering, which enables the differentiation of human induced pluripotent stem (iPS) cells into mature podocytes with more than 90 percent efficiency.

Linking the differentiation process with organ-on-a-chip technology pioneered by his team, the researchers went on to engineer the first in vitro model of the human glomerulus, demonstrating effective and selective filtration of blood proteins and podocyte toxicity induced by a chemotherapy drug in vitro.

Ingber is also theJudah Folkman Professor of Vascular Biologyat Harvard Medical School (HMS) and the Vascular Biology Program at Boston Childrens Hospital.

The development of a functional human kidney glomerulus chip opens up an entirely new experimental path to investigate kidney biology, carry out highly personalized modeling of kidney diseases and drug toxicities, and the stem cell-derived kidney podocytes we developed could even offer a new injectable cell therapy approach for regenerative medicine in patients with life-threatening glomerulopathies in the future, said Ingber.

Ingbers team has engineered multiple organs-on-chips that accurately mimic human tissue and organ-level physiology. These platforms are currently being evaluated by the Food and Drug Administration (FDA) as a tool to more effectively study the effects of potential chemical and biological hazards found in foods, cosmetics or dietary supplementsthan existing culture systems or animal models. In 2013, his team developed an organ-on-a-chip microfluidic culture device that modeled the human kidneys proximal tubule, which is anatomically connected to the glomerulus and salvages ions from urinary fluid.

Now, with the teams newly engineered human kidney glomerulus-on-a-chip, researchers also can get in vitro access to the kidneys core filtration mechanisms that are critical for drug clearance and pharmacokinetics, in addition to studying human podocytes at work.

To generate almost pure populations of human podocytes in cell culture, Samira Musah, the studys first author and HMS Deans Postdoctoral Fellow who is working with Ingber at the Wyss Institute, leveraged pieces of the stem cell biologists arsenal, and merged them with snippets taken from Ingbers past research on how cells in the body respond to adhesive factors and physical forces in their tissue environments.

Our method not only uses soluble factors that guide kidney development in the embryo but, by growing and differentiating stem cells on extracellular matrix components that are also contained in the membrane separating the glomerular blood and urinary systems, we more closely mimic the natural environment in which podocytes are induced and mature, said Musah. We even succeeded in inducing much of this differentiation process within a channel of the microfluidic chip, whereby applying cyclical motions that mimic the rhythmic deformations living glomeruli experience due to pressure pulses generated by each heartbeat, we achieve even greater maturation efficiencies.

The complete microfluidic system closely resembles a living, three-dimensional cross-section of the human glomerular wall. It consists of an optically clear, flexible, polymeric material the size of a computer memory chip in which two closely opposed microchannels are separated by a porous, extracellular matrix-coated membrane that corresponds to the kidneys glomerular basement membrane. In one of the membrane-facing channels, the researchers grow glomerular endothelial cells to mimic the blood microvessel compartment of glomeruli. The iPS cells are cultured on the opposite side of the membrane in the other channel that represents the glomerulus urinary compartment, where they are induced to form a layer of mature podocytes that extend long cellular processes through the pores in the membrane and contact the underlying endothelial cells. In addition, the devices channels are rhythmically stretched and relaxed at a rate of one heart beat per second by applying cyclic suction to hollow chambers placed on either side of the cell-lined microchannels to mimic physiological deformations of the glomerular wall.

This in vitro system allows us to effectively recapitulate the filtration of small substances contained in blood into the urinary compartment while retaining large proteins in the blood compartment just like in our bodies, and we can visualize and monitor the damage inflicted by drugs that cause breakdown of the filtration barrier in the kidney, said Musah.

The study was also co-authored by Wyss Institute Core Faculty member George Church, who also is Professor of Genetics at HMS and Professor of Health Sciences and Technology at Harvard and the Massachusetts Institute of Technology (MIT), and who served as a co-mentor of Musah with Ingber. Other authors include Akiko Mammoto and Tadanori Mammoto, who at the time of the study were Instructors in the Vascular Biology Program and Department of Surgery at Boston Childrens Hospital, as well as Thomas Ferrante, Sauveur Jeanty, Kristen Roberts, Seyoon Chung, Richard Novak, Miles Ingram, Tohid Fatanat-Didar, Sandeep Koshy, and James Weaver.

Funding for the study was provided by the Defense Advanced Research Projects Agency (DARPA). Musah was supported by a HMS Deans Postdoctoral Fellowship, Postdoctoral Enrichment Program Award from the Burroughs Wellcome Fund, UNCF-Merck Postdoctoral Fellowship, and an NIH/NIDDK Nephrology Training Grant.

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Engineering human stem cells to model the kidney's filtration barrier on a chip - Harvard School of Engineering and Applied Sciences

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Oxford Genetics has licensed CRISPR gene editing technology from ERS Genomics. The agreement gives the British synthetic biology service provider the right to use CRISPR technology to improve gene therapy viral vectors and develop cell lines.

Oxford, United Kingdom-based Oxford Genetics has secured the nonexclusive rights to the CRISPR intellectual property. Oxford Genetics plans to use the technology to provide genome engineering services and support its cell line development and gene therapy viral vector R&D efforts. The agreement also clears Oxford Genetics to use the CRISPR-edited cells lines in the production of biotherapeutics. And to use CRISPR to develop research tools and reagents for sale.

News of the agreement comes almost exactly three years after Horizon Discovery licensed CRISPR intellectual property from ERS Genomics for use in similar applications. The nonexclusive deal between ERS Genomics and Horizon Discoverywhich is based 70 miles away from Oxford Genetics in Cambridgegave the genomics research business the right to use CRISPR to develop research tools, kits and reagents and in other applications.

ERS Genomics was cofounded by Emmanuelle Charpentier, Ph.D., one of the key players in the story of the discovery of the CRISPR-Cas9 immune system and its role in cleaving DNA. Charpentier set up the organization to facilitate access to the CRISPR-Cas9 intellectual property she holds. The firm is on the same side of the CRISPR patent dispute as CRISPR Therapeutics, Intellia Therapeutics and Caribou Biosciences. Together, the companies are appealing the U.S. patent boards ruling in the Broad Institute case.

The ruling looked at the question of whether the it was obvious to apply CRISPR to eukaryotic cells, such as the CHO and HEK293 cell lines Oxford Genetics uses in its cell line development services. But the uncertainty created by the ongoing patent dispute has not stopped Oxford Genetics from striking a deal to add CRISPR to its arsenal.

Licensing the CRISPR gene editing technology from ERS Genomics is another step on our journey to establishing the most efficient and integrated service portfolio in this sector. We are excited to be adding this technology to our existing portfolio in the synthetic biology space and supporting the rapidly expanding market for products and services that utilise genome engineering technologies, Paul Brooks, Ph.D., chief commercial officer at Oxford Genetics, said in a statement.

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Oxford Genetics licenses CRISPR tech to power synbio push - FierceBiotech

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Brammer Bio says its commercial-scale gene therapy manufacturing facility in Cambridge, Massachusetts, US, will open in the second half of 2017.

The contract development and manufacturing organization (CDMO) said the site offers process development, clinical phase, and current Good Manufacturing Practices (cGMP) services for cell and gene therapies.

We are delighted to add an experienced commercial biologics team and facilities to help meet the needs of this transformative industry, said Mark Bamforth, CEO of Brammer.

Brammer has completed a Type-C meeting with the US Food and Drug Administration (FDA) to assess the plans for the Massachusetts-based site. A Type-C meeting regards the development and review of drugs or biological drug products regulated by the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

A Site Master File will be submitted later this year to support Brammers clients product applications.

The site originally housed Biogens clinical and commercial biologics manufacturing facility.

Brammer purchased it along with Biogens distribution center in Somerville, Massachusetts, on January 1, 2017.

The Somerville site offers Brammer nearby storage and distribution capabilities.

The announcement follows Brammers 2016 plans to renovate its 50,000 square-foot facility in Lexington, Massachusetts, to cater for late stage and commercial therapy supply.

Brammers facilities now offer 230,000 square feet of development, distribution and cGMP manufacturing capabilities across Florida and Massachusetts, US.

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Brammer invests in commercial-ready gene therapies - OutSourcing-Pharma.com

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Being acquainted with what each time in your cycle means and the accompanying symptoms can help you better understand your period.

Lets start by establishing how we count the days. Day 1 is the first full day of flow. The length of your cycle is counted from Day 1 of this cycle and until Day 1 of your next cycle.

Cycles vary widely anything between 24 to 35 days is perfectly normal, but for simplicity sake, we often refer to a 28-day cycle. (This is especially applicable if youre on birth control that works on a 28-day cycle.)

More:These Two Life Events Could Predict Early Menopause

Your cycle has two major phases, follicular and luteal, with a brief but crucial third phase, ovulation, in the middle. When it comes to your menstrual cycle, estrogen and progesterone are the primary hormone drivers. Fun fact: Its what many forms of the birth control pill consist of. These hormones are secreted by the ovary, and are responsible for stimulating the lining of the uterus or womb, called the endometrium, to grow and prepare for a potential pregnancy.

The first phase of your cycle is called the follicular phase. This is because the main event during this phase is the creation of a follicle in the ovary. It is from this follicle that an egg is released, which is known as ovulation. This phase varies in length, but is usually between 12 and 16 days.

During your period in the first few days of your cycle, both estrogen and progesterone are low. Starting around Day 4 to 5, estrogen levels gradually begin to rise and what is called a dominant follicle forms. The dominant follicle prepares the egg for ovulation. Around cycle Day 10, estrogen production increases dramatically and progesterone slowly begins to rise. Estrogen surges around Day 12 to 13, meaning it shoots up, quadrupling levels, but then falls almost all the way back to pre-surge levels.

Twenty-four to 36 hours after estrogen surge, ovulation occurs. The dominant follicle releases an egg and becomes what is called the "corpus luteum." In a 28-day cycle, this is around Day 14.

More: "Period in a Petri Dish" Could Have a Big Impact on Reproductive Research

The last phase of your cycle, which is called the luteal phase referring to the corpus luteum, begins after ovulation. This part of your cycle is less variable in length and should consistently be 13 to 14 days. Both estrogen and progesterone rise sharply 1 or 2 days after ovulation. They peak about a week later around Day 20 to 22 and then if pregnancy does not occur, fall precipitously, resulting in low levels by Day 28. Now that your hormone levels are low, your next period starts, and it all begins again.

As you can see, there are some hormonal highs and lows throughout the month. Considering what is happening with your hormones may help to explain why some days you feel awesome and others not so much. For instance,according to Sarah Jio at Womans Day,during the first week of your cycle when estrogen levels are low, you may have less energy and a lower libido. So prepare for that. Plan for extra sleep if youre tired or extra time with your partner if you notice your libido is low.

Menstrual migraines are a classic example. According to the Mayo Clinic, estrogen and progesterone may affect brain chemicals associated with headaches. Drops in estrogen, which occur both around the time of ovulation as well as premenstrually, can make headaches worse. Birth control pills can control headaches by keeping estrogen levels steady.

Although the exact causes are not known,the Office on Womens Health states that an important cause of PMS is changing levels of hormones. Since PMS symptoms can begin anywhere from 1 to 2 weeks before your period starts, consider what is happening with estrogen and progesterone during that time. They are both rising dramatically and then falling precipitously. No wonder some women just dont feel well during that hormonal roller-coaster ride.

Every woman thinks that its the sudden drop in estrogen from not ovulating that causes the problems. But in reality, its the fluctuation of estrogen along with less progesterone that is behind many of the typical symptoms of perimenopause,says Dr. Steven R. Goldstein, professor of obstetrics and gynecology at NYU Medical Center in New York City.

More: Irregular Periods Explained: 8 Big Reasons Your Period's Gone MIA

Understanding the elaborate hormonal changes that occur throughout the month and in what way they may affect how you feel including your mood, energy and libido to name a few can help you anticipate and prepare for the highs and lows. This way, you can ride the hormonal wave instead of being crushed by it.

By Katie Killoran

Originally published on HelloFlo.

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Here's What Happens to Your Hormone Levels During Your Period - SheKnows.com

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Neurology Advisor

In Hypogonadism, Stroke May Be Prevented With Testosterone Replacement Therapy Neurology Advisor Patients with primary hypogonadism, secondary hypogonadism related to overt hypothalamic pituitary pathology, HIV infection, metastatic cancer, a history of prostate cancer, prostate specific antigen >4 ng/mL, elevated hematocrit, or a history of ...

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In Hypogonadism, Stroke May Be Prevented With Testosterone Replacement Therapy - Neurology Advisor

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Q: Ive been reading a lot about stem cells recently. Willstem cell research change the treatment of heart disease?

A: Theres some exciting early data where scientists have been able to use stem cells for regeneration of cardiac tissue, in particular certain parts of the heart or maybe even an entire heart in mice or rats.

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However, its not been done yet in humans reliably and that would be the next step. If the research bears out, we may see this as an option for heart patients in perhaps five to 10 years.

The area where stem cells might first be used is in patients who have had damage to their heart because of a heart attack. These patients have scarring on the heart and that area of the heart is not beating anymore. If we can regenerate cardiac tissue to replace this scarred tissue, the hope is to get the heart fully working again.

Growing whole new hearts will likely be later down the line and will depend on the success of the research.

Preventive cardiologistHaitham Ahmed, MD, MPH

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Will Stem Cell Research Change Treatment of Heart Disease? - Health Essentials from Cleveland Clinic (blog)

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Kidney research at the University of Virginia School of Medicine has unexpectedly led to a discovery about the formation of the heart, including the identification of a gene responsible for a deadly cardiac condition.

UVA scientists were surprised to discover that the heart's inner lining forms from the same stem cells, known as "precursor cells," that turn into blood. That means a single type of stem cell turns into both our blood and a portion of the organ that will pump it.

The researchers determined that a particular gene, S1P1, is vital for the proper formation of the heart. Without it, the heart tissue produced by the precursor cells develops a sponginess that compromises the heart's ability to contract tightly and pump blood efficiently. In people, that is known as ventricular non-compaction cardiomyopathy, a dangerous condition that often leads to early death.

"Many patients who suffer from untreatable chronic diseases, including heart and kidney diseases, are in waiting lists for limited organ transplantation. Therefore, there is an urgent need to understand what happens to the cells during disease and how can they be repaired," said researcher Yan Hu, PhD. "Every organ is a complex machine built by many different cell types. Knowing the origin of each cell and which genes control their normal function are the foundations for scientists to decipher the disease process and eventually to find out how to guide the cells to self-repair or even to build up a brand new organ using amended cells from the patients."

Far-Reaching Consequences

The researchers, led by Maria Luisa S. Sequeira-Lopez, MD, of UVA's Child Health Research Center, were investigating how the kidney forms when they noted that the deletion of the S1P1 gene in research mice had deadly consequences elsewhere in the body. "We were studying the role of these genes in the development of the vasculature of the kidney," she recalled. "The heart is the first organ that develops, and so when we deleted this gene in these precursor cells, we found that it resulted in abnormalities of the heart, severe edema, hemorrhage and low heart rate."

That led them to look more closely at the heart. It was then that they discovered the gene deletion had caused thin heart walls and other cardiac problems in developing mice embryos. "So then we had to study the heart when the kidneys were still not even formed," she said. "We had to go far outside our comfort zone."

Their findings would prove unexpected even for scientists who specialize in the development of the heart. "For a long time, scientists believed that each organ developed independently of other organs, and the heart developed from certain stem cells and blood developed from blood stem cells," explained researcher Brian C. Belyea, MD, of the UVA Children's Hospital. "A number of studies done in this lab and others, including this work, shows that there's much more plasticity in these precursor cells. What we found is that cardiac precursor cells that are present in the embryonic heart do indeed give rise to components of the heart in adults but also give rise to the blood cells."

The researchers were so surprised by their discovery that they went back and validated their findings repeatedly, using multiple techniques, including new techniques that they developed.

Belyea said that the discovery about the important role of the S1P1 gene may one day lead to better treatments for that condition. "We hope," he said, "that this is a stepping stone for our clinical colleagues."

Story Source:

Materials provided by University of Virginia Health System. Note: Content may be edited for style and length.

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Kidney research leads to surprising discovery about how the heart forms - Science Daily

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Lab-engineered bone (the outer layer) with functional bone marrow (the inner layer). Image: Varghese Lab at UC San Diego

Thanks to advances in medicine, bone marrow transplants are no longer the last resorts they once were. Every year, thousands of marrow transplants are performed, a common treatment for ailments from bone marrow disease to leukemia. But because they first require a patient undergo radiation to kill off any existing bone marrow stem cells, marrow transplants remain incredibly hard on a patient.

Now, engineers at the University of California San Diego have developed a synthetic bone implant with functional marrow able to produce its own blood cells. So far, researchers revealed in a paper published in the Proceedings of the National Academy of Sciencesthis week, they have successfully tested the engineered bone tissues in mice. But one day, those biomimetic bone tissues could provide new bone marrow for human patients in need of transplants, too.

The implant does away with the need for radiation by giving donor cells their own space in the body to grow. Inside the implant, there is no threat of those cells being overtaken by the bodys native stem cells.

In mice, the researchers implanted the synthetic bone tissues with functional marrow under the skin. After six months, those donor cells were still alive and had begun supplying the mice with new blood cells.

The implants were designed to replicate the long bones in the body, with an outer bone compartment containing calcium phosphate minerals to build bone cells, and an inner area for donor stem cells that produce blood cells.

When implanted, they grew into bone tissues with working blood vessel network and functional bone marrow that supplied the body new blood cells. After 24 weeks, researchers found a mix of host and donor blood cells was still circulating in the bloodstream of the mice.

A treatment based on this technology would only work for patients with non-malignant bone marrow diseases, like aplastic anemia, a condition where the body cant make enough platelets and blood cells. Thats because while the technique can replenish types of cells that are lacking, it cant doing anything to fight off cells that have mutated and are spreading. Cancer patients would still need need to undergo radiation therapy to have their cancerous cells wiped out.

Much more research is needed, of course, before these implants are ready to make their way into human patients. But whats exciting here is that the synthetic bone tissues were not only functional, they allowed donor marrow to grow and survive for many weeks in the presence of host cells, and for the products of that marrow to make their way into the bodys circulatory system. Pretty neat.

See the article here:
This Synthetic Bone Implant Could Replace Painful Marrow Transplants - Gizmodo

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Sensational 8-Year-Old Violinist Living With Painful Disease

WINSTON-SALEM, NC Its hard to walk through life without hitting a sour note or two. In Winston-Salem, there's a young boy with talent beyond his years and a disease that nearly crippled him. His father gave up his career to take care of his son and to get him healthy.

Child Prodigy

We only listen to classical music at home," said Lucas Sant, a father of three living in Winston-Salem. He sits with his youngest, Helen, 2, on his lap. His second oldest daughter, Maria-Anita, 7, sits on his right and his only son, Caesar, 8, sits to his left.

Hes telling WFMY News 2s reporter, Hope Ford, about his sons remarkable talent.

When he was just a baby, we bought Baby Einstein, and you know, they have the animals and the music. So, we bought him a little toy piano, Lucas began. And one day, when he was seven months old, we heard this music coming from the room. It sounded like the toy piano, but it was the music from the Baby Einstein.

Lucas turned to his wife, Aline, with a knowing smile and said, We have our work to do with this boy.

Videos uploaded to YouTube, show a baby Caesar, waving his arms along to classical music such as Beethoven, almost as if he were conducting a symphony.

A baby Caesar and his father listening to classical music. (Photo: Sant family)

Violin lessons started the age of two.*

He started playing Vivaldi. He would pick up things very quick, said Lucas. Everybody was very impressed.

GoFundMe

All the Sant children are homeschooled and it would be no surprise to learn Caesar is just as brilliant with a pencil as he is with an instrument. The young boy is ahead in math and other subjects and earned a black belt in karate at 5-years-old.

A Painful Disease

Lucas sat in his seat, as baby Helen decided she wanted to leave the room to see what her mom was up to. As she ran into the next room, Lucas continued his story.

Immediately, he started to get sick. Before five, he had the first stroke.

Caesar has sickle cell anemia.

You never know anything until you experience, Lucas said in a soft voice.

Sickle cell anemia is a blood disease. Normal red blood cells are round and flexible to carry oxygen throughout the body. Caesars blood cells are sickle-shaped or bent and get stuck, slowing the flood of blood and oxygen.

Lucas explained, Its different. Its my son and I never seen this thing.

Caesar, who up until this point sat quietly next to his father with his violin in his lap said, I feel bad. I dont feel good when Im sick.

The curly haired violinist has three strokes before the age of six. The first two left his arms weak, but he rebounded, performing the National Anthem at the Grasshoppers Game in 2013.

The third one was a different stroke, said his dad.

Caesar lost feeling in his arms and legs after his third stroke, leaving him partially paralyzed for nearly six months.

At first, even his eyes was not moving. But, when he did wake up, all of a sudden your son not walk, not run, not stand up, Lucas said as if he was still trying to make sense of it all.

Doctors told the Sant family, It is very unlikely your son is going to die but do not expect much from him.

Lucas paused for a moment and continued, But the good thing there, you really meet God. What am I supposed to do God? Please tell me.

The only thing that seemed right at the time, was for Lucas to give up his career. The father of three was a neuroscientist at Wake Forest Baptist Medical Center.

Forget about my life. I said, Im going give my life to this boy.

Young Caesar in the hospital. (Photo: Sant family)

The Sant family built a small play gym in the basement of their home. Here, Lucas would help Caesar with physical therapy, as they could not afford to hire someone full time to help him regain strength and movement in his arms and legs.

Some days and good and some are bad. Three years after his last stroke, Caesar still winces in pain as he goes through his exercises. But, he finds moments to laugh with his siblings, who cheer him on. And as an 8-year-old, he is a little hard to get under control. For Lucas, the physical therapy takes a toll on his as well.

First, Im not a physical therapist. I have a lot of patience but its very hard for you see your son one way, said Lucas. Sometime, we have to take breaks because it is difficult and it sometimes weighs on my own health.

But, once again, Caesar regained his strength, returning to the Grasshoppers stadium in 2017 to perform the National Anthem once again.

A Small Miracle

Every month, Caesar and his family travel to Charlotte for blood transfusions to lower the risk of Caesar having another stroke. He'll have to do this for the unforeseeable future and there are risks.*

Frequent blood transfusions can lead to iron overload which is sometimes fatal. Caesar's family is trying for a bone marrow transplant which has a higher percentage of curing his sickle cell disease.

They have a donor- his baby sister, Helen.

As if she knew her name had been mentioned, the young girl, called the boss of the family, walked back into the room, sharing bites of her rice with her siblings and father.

Lucas and his wife wanted another child, but they also wanted to ensure the next child would not have the sickle cell anemia trait. they also wanted to ensure they would have a 100 percent genetic match for Caesar's procedure.

Maria-Anita was also born with sickle cell anemia, but unlike her brother, has yet to experience any complications.

So, Aline got pregnant via in vitro fertilization. Doctors only planted cells that were a genetic match and only healthy cells were selected. Thus, Helen was conceived and at birth, her umbilical cord was collected.

Helen, was born sickle-cell free.

They took the stem cells from the umbilical cord and now they have perfect cells, to do the transplant on him, said Lucas.

The Next Step

The Sant family is trying to raise money for a bone marrow/stem cell transplant. The process is long and costly. According to Johns Hopkins, one hospital that specializes in bone marrow/stem cell transplants, they say the cost can run as high as $500,000.

However, sickle cell anemia can be cured with the procedure.

Offering her big brother another big of food, Helen, Caesars sisterly hero, smiled and ran off.

Lucas continued to explain the familys financial situation.

Its difficult, with me not having a job. But, we have had people help us along the way. But, we are still trying so hard to raise money for the surgery.

A GoFundMe account was started in 2013. To date, $38,000 has been raised. The family also started a website to give updates and sell merchandise to help raise funds as well.

Caesar still walks with a limp and must be careful when sitting down. Lucas looked at his son and said Were so happy because he got back. He got back, but the job is not done. Faith, hope, these things so real. Cause if dont have what you can do? You give up right there.

Caesar piped in again, Sometimes I tell my father, papa, I dont know when Im going to be back, but God is always with me.

Lucas isnt giving up. His hope, to have son healthy by 2018.

And Caesars hope?

I want to be a musician and a conductor.

*This story has been updated to correct information. Lessons for Caesar started at the age of 2 and 300ml of his blood is replaced every month during his blood transfusions.

5 Facts About Sickle Cell Disease (CDC)

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Sensational 8-Year-Old Violinist Living With Painful Disease | KSDK ... - KSDK

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Boston Business Journal

Cambridge gene editing firm CRISPR to use delivery tech honed ... Boston Business Journal Cambridge gene editing biotech CRISPR Therapeutics has grabbed an exclusive license from MIT to use a delivery system that could make such treatments ... -$0.51 Earnings Per Share Expected for Crispr Therapeutics AG (CRSP) This QuarterThe Cerbat Gem Crispr Therapeutics AG (CRSP) Given Average Recommendation of "" by AnalystsTranscript Daily Rodger Novak Sells 49384 Shares of Crispr Therapeutics AG (CRSP) StockSports Perspectives Chaffey Breeze -Key Gazette -Petro Global News 24 all 12 news articles »

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Cambridge gene editing firm CRISPR to use delivery tech honed ... - Boston Business Journal

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Science

We see it all the time in movies and TV shows. Someone is frozen and brought back to life. Its called cryonics. Is there any actual science behind it?

Published on February 21, 2011

We see it all the time in movies. A person gets frozen or put in cryosleep and then unfrozen at a later date with no aging taking place, or other ill effects.

Sometimes this happens on purpose, like to someone with an incurable disease hoping a cure exists in the future, or sometimes by accident, like someone getting frozen in a glacier.

The science behind it does exist and the application of the practice is called cryonics. Its a technique used to store a persons body at an extremely low temperature with the hope of one day reviving them. This technique is being performed today, but the technology behind it is still in its infancy.

Someone preserved this way is said to be in cryonic suspension. The hope is that, if someone has died from a disease or condition that is currently incurable, they can be frozen and then revived in the future when a cure has been discovered.

Its currently illegal to perform cryonic suspension on someone who is still alive. Those who wish to be cryogenically frozen must first be pronounced legally dead which means their heart has stopped beating. Though, if theyre dead, how can they ever be revived?

According to companies who perform the procedure, legally dead is not the same as totally dead. Total death, they claim, is the point at which all brain function ceases. They claim that the difference is based on the fact that some cellular brain function remains even after the heart has stopped beating. Cryonics preserves some of that cell function so that, at least theoretically, the person can be brought back to life at a later date.

After your heart stops beating and you are pronounced legally dead, the company you signed with takes over. An emergency response team from the facility immediately gets to work. They stabilize your body by supplying your brain with enough oxygen and blood to preserve minimal function until you can be transported to the suspension facility. Your body is packed in ice and injected with an anticoagulant to prevent your blood from clotting during the trip. A medical team is on standby awaiting the arrival of your body at the cryonics facility.

After you reach the cryonics facility, the actual freezing can begin.

They could, and while youd certainly be frozen, most of the cells in your body would shatter and die.

As water freezes, it expands. Since cells are made up of mostly water, freezing expands the stuff inside which destroys their cell walls and they die. The cryonics companies need to remove and/or replace this water. They replace it with something called a cryoprotectant. Much like the antifreeze in an automobile. This glycerol based mixture protects your organ tissues by hindering the formation of ice crystals. This process is called vitrification and allows cells to live in a sort of suspended animation.

After the vitrification, your body is cooled with dry ice until it reaches -202 Fahrenheit. After this pre-cooling, its finally time to insert your body into the individual container that will be placed into a metal tank filled with liquid nitrogen. This will cool the body down to a temperature of around -320 degrees Fahrenheit.

The procedure isnt cheap. It can cost up to $200,000 to have your whole body preserved. For the more frugal optimist, a mere $60,000 will preserve your brain with an option known as neurosuspension. They hope the technology in the future will allow them to clone or regenerate the rest of the body.

Many critics say the companies that perform cryonics are simply ripping off customers with the dream of immortality and they wont deliver. It doesnt help that the scientists who perform cryonics say they havent successfully revived anyone, and dont expect to be able to do so anytime soon. The largest hurdle is that, if the warming process isnt done at exactly the right speed and temperature, the cells could form ice crystals and shatter.

Despite the fact that no human placed in a cryonic suspension has yet been revived, some living organisms can be, and have been, brought back from a dead or near-dead state. CPR and Defibrillators can bring accident and heart attack victims back from the dead daily.

Neurosurgeons often cool patients bodies so they can operate on aneurysms without damaging or rupturing the nearby blood vessels. Human embryos that are frozen in fertility clinics, defrosted and implanted in a mothers uterus grow into perfectly normal human beings. Some frogs and other amphibians have a protein manufactured by their cells that act as a natural antifreeze which can protect them if theyre frozen completely solid.

Cryobiologists are hopeful that nanotechnology will make revival possible someday. Nanotechnology can use microscopic machines to manipulate single atoms to build or repair virtually anything, including human cells and tissues. They hope one day, nanotechnology will repair not only the cellular damage caused by the freezing process, but also the damage caused by aging and disease.

Some cryobiologists have predicted that the first cryonic revival might occur as early as year 2045.

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Can A Human Be Frozen And Brought Back To Life? - Zidbits

Recommendation and review posted by Bethany Smith