Heart Disease: The Genetics Behind It.
You got your brown eyes from your dad. You #39;re a lefty thanks to your mom. But you also may be at greater risk for heart disease because of your inheritance a...

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CSA genetics polycystic kidneys
CSA genetics case done well - showing you don #39;t need to mention chromosomes, genes etc.

By: Matthew Smith

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Trans Ova Genetics - 2015 Denver Display Video
Trans Ova Genetics provides industry-leading reproductive technologies and expertise to cattle breeders through a unique professional services team that work...

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Project Mineoid | E3 | Advanced Genetics!
Project Mineoid is a new SMP server created by CynicaGaming. It started out as an idea on reddit and took form a little while after that. Join us in our jour...

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Bagito: Genetics Explained
Andrew believes that Albie is his real child because they really are look alike. Subscribe to ABS-CBN Entertainment channel! - http://bit.ly/ABS-CBNEntertain...

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Genetics- Unprofessional Psychopaths (prod. by MainPiece)
Follow on instagram: genetics4 Watch in 1080p.

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Human evolution questioned: 'Big brain gene found humans, not chimps'

A single gene may have paved the way for the rise of human intelligence by dramatically increasing the number of brain cells found in a key brain region.

This gene seems to be uniquely human: It is found in modern-day humans, Neanderthals and another branch of extinct humans called Denisovans, but not in chimpanzees.

By allowing the brain region called the neocortex to contain many more neurons, the tiny snippet of DNA may have laid the foundation for the human brain's massive expansion.

"It is so cool that one tiny gene alone may suffice to affect the phenotype of the stem cells, which contributed the most to the expansion of the neocortex," said study lead author Marta Florio, a doctoral candidate in molecular and cellular biology and genetics at the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden, Germany.

Still, it's likely this gene is just one of many genetic changes that make human cognition special, Florio said.

An expanding brain

The evolution from primitive apes to humans with complex language and culture has taken millions of years.

Some 3.8 million ago, Australopithecus afarensis, the species typified by the iconic early human ancestor fossil Lucy, had a brain that was less than 30 cubic inches (500 cubic centimeters) in volume, or about a third the size of the modern human brain.

By about 1.8 million years ago, Homo erectus was equipped with a brain that was roughly twice as big as that of Australopithecus.

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Human evolution questioned: 'Big brain gene found humans, not chimps'

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Chicago, IL (PRWEB) February 27, 2015

Muscular Dystrophy Association research grantee Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University, recently announced a potentially game-changing advance in gene modification for boys and young men with Duchenne muscular dystrophy (DMD). The results were published Feb. 18 in Nature Communications, and Gersbach will discuss their implications at MDA's 2015 Scientific Conference, to be held March 11-14 in Washington, D.C.

Background: Gersbach and team are investigating a new genetic therapeutic technique that has the potential to treat more than half of patients fighting DMD and could be developed as a permanent, one-time treatment. Known as CRISPR-Cas9 genome editing, the strategy targets a large area of the dystrophin gene (DNA) and is designed to cause production of shorter-than-normal, but still functional, dystrophin protein in muscle tissue. If successful in humans, it could prolong function and increase longevity.

Other promising experimental DMD treatments such as the experimental drugs eteplirsen and drisapersen are currently being tested in clinical trials, but because these other treatments target dystrophin RNA rather than DNA, they would not result in a permanent correction and would require repeated treatment over a patients lifetime.

Ever since MDA-supported researchers identified flaws in the dystrophin gene as the cause of DMD in 1986, the organization has been in the forefront of DMD research, while continuing its commitment to ongoing patient care. One in three DMD patients in the U.S. attends an MDA-supported clinic, and thanks to MDA-funded laboratory and clinical investigations, there are now at least eight experimental compounds in clinical trials for DMD.

Statement from Grace Pavlath, Ph.D., MDA Senior Vice President & Scientific Program Director: "MDA is proud to have supported Dr. Gersbach for the development of this game-changing advance. This is another prime example of our commitment to making urgently-needed progress for those affected by muscular dystrophy and related life-threatening diseases. While we have every hope that other drugs currently in late-stage development for DMD will prove to be safe and effective, it is important to support multiple strategies in the development pipeline as MDA continues to search for treatments and cures."

About MDA

The Muscular Dystrophy Association is the worlds leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

Visit mda.org and follow us at facebook.com/MDAnational and @MDAnews. Learn more about MDA's mission by watching this video.

Claire Orphan Media Relations Manager (312) 260-5928 corphan(at)mdausa(dot)org

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Statement: New MDA-Funded Genetic Therapy Technique Targets DMD

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CHOP study of common variable immunodeficiency finds gene crucial to immune defense

IMAGE:Dr. Hakon Hakonarson is the director of the Center for Applied Genomics of The Children's Hospital of Philadelphia. view more

Credit: The Children's Hospital of Philadelphia

Genomics researchers analyzing a rare, serious immunodeficiency disease in children have discovered links to a gene crucial to the body's defense against infections. The finding may represent an inviting target for drugs to treat common variable immunodeficiency (CVID).

A team led by Hakon Hakonarson, M.D., Ph.D., director of the Center for Applied Genomics at The Children's Hospital of Philadelphia (CHOP), reported their findings online Feb. 10 in the Journal of Allergy and Clinical Immunology.

The researchers found 11 single nucleotide polymorphisms (SNPs) on the 16p11.2 locus of chromosome 16. SNPs are changes in one letter of DNA, compared to the more typical sequence at a given location. Of particular interest, the study team found variants in the gene ITGAM, carrying codes for an integrin protein, which regulates cellular contact and adhesion.

"This association is of high biological relevance, because ITGAM plays an important role in normal immune responses," said Hakonarson. "Other researchers have shown that mice in which this gene has been knocked out have immune deficiencies." He added that his team's findings may have broader implications for patients who do not have these specific rare variants, because the integrin protein affects many important pathways in immune function.

A child with CVID has a low level of antibodies, reducing the body's ability to fight disease, and leaving the child vulnerable to recurrent infections. CVID can first occur early or later in life, and the symptoms are highly variable. Frequent respiratory infections may lead to permanent lung damage. Patients may also suffer joint inflammation, stomach and bowel disorders, and a higher risk of cancers.

The great variability of the disease and the lack of a clear-cut diagnostic test often mean that CVID goes undiagnosed for years before doctors can begin treatment.

The study team performed an association analysis that focused on immune-related genes in a cohort of 360 CVID patients and 21,610 healthy controls. They used a genotyping chip specialized to search for gene variants previously implicated in autoimmune and inflammatory diseases, to pinpoint SNPs associated with CVID.

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Novel gene variants found in a difficult childhood immune disorder

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Genetics Affects Your Destiny
People say you can do whatever you want and you can be whatever you want as long as you put your mind into it. But is this statement really true? Or is it pr...

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Genetics In Genesis
What is DNA? What does our DNA structure tell us about our origins? Do we have a common ancestor? If so, who or what is it? Is macroevolution provable through our genetic makeup? The book of...

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OG RASKAL GENETICS AT CANNABIS CUP 2015
PAYASOGROW TOOK @GUAPCITY IN TO CANNABIS CUP 2015 FOR A LOOK INTO THE OG RASKAL GENETICS BOOTH AND TO TRY OUT SOME WHITEFIRE TAKE A LOOK.

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Carlsbad, CA (PRWEB) February 28, 2015

A study out of the University of Oxford has developed a promising new gene therapy procedure for the treatment of a hereditary ocular condition that leads to progressive blindness in patients. Authored by Dr. Robert MacLaren, professor of ophthalmology at the University of Oxford and published in the Lancet Medical Journal on January 16, 2014, the study treated six patients suffering from choroideremia, a rare genetic disorder that mostly affects men and leads to the degeneration of the choroid and retina, eventually causing blindness. Night blindness and loss of peripheral vision are the most common symptoms of choroideremia. For the study, researchers injected the subjects with healthy genetic material in an attempt to repair the damaged portions of the patients own gene, halting the onset of blindness.

Developing technologies and gene therapy are the future of ophthalmology, said Carlsbad LASIK surgeon Dr. Michael Tracy. In the past, there was little that could be done for certain patients facing blindness due to genetic and degenerative conditions. Gene therapy can lead to treatments that can stop the onset of blindness in certain patients before its too late.

For the Oxford study, researchers used virus particles to add the genetic material to the fluid behind the retina. The goal of the therapy was for the new genetic material to fix or patch flaws in the patients damaged DNA. Timing was an important element of the study. The participants in the study were all treated before their visual acuity had been irreversibly diminished, giving the therapy a greater chance of success. Of the six participants, two reported significant improvement in visual acuity, the other four reported improvement in night vision and maintained the same level of visual acuity as before the treatment in the affected eye. The therapy does involve the risk of further or permanent damage as it requires detachment of the retina, but none of the participants reported further degeneration of visual acuity in the treated eye.

This research is very important because it can pave the way for more gene therapy studies for treating other debilitating conditions that can lead to blindness, such as macular degeneration, added Dr. Tracy.

Dr. Michael Tracy is a board-certified ophthalmologist in Carlsbad, CA. He completed his residency training in ophthalmology at the Scheie Eye Institute at the University of Pennsylvania and his fellowship in Corneal and Refractive Surgery at the Bascom Palmer Eye Institute. Dr. Tracy specializes in LASIK and PRK surgery at his private practice at Carlsbad Eye Care.

To contact Carlsbad Eye Care, please visit http://carlsbad-eye-care.com/ or call (760) 603-9910.

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New Gene Therapy Procedure Could Hold Key to Preventing Blindness for Certain Patients

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Pioneering Longevity Researcher Insilico Medicine Takes Top Prize at PMWC 2015
As billions pour into biotech and medical research firms looking for life-extending medications, venture capitalists at the Personalized Medicine World Confe...

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The phases of a clinical trial - Virtual Clinical Trials: Spinal Cord Injury
In this movie clip, students learn about clinical trial phases. This information is reviewed prior to setting up the spinal cord injury clinical trial.

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Analyzing results and drawing conclusions - Virtual Clinical Trials: Spinal Cord Injury
In the Virtual Clinical Trials simulations, results from the clinical trials are analyzed in order to draw conclusion about the medical advancements being te...

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Report Details

Translational Regenerative Medicine - new study showing you trends, R&D progress, and predicted revenues Where is the market for regenerative medicine heading? What are the commercial prospects for this market and related technologies? Visiongain's brand new report shows you potential revenues and other trends to 2025, discussing data, opportunities and prospects.

Visiongain's report lets you assess regenerative medicine: cell-based therapies that aim to restore function and regenerate diseased tissues. Our 260 page report provides 145 tables, charts, and graphs. Discover the most lucrative areas in the industry and the future market prospects. Our new study lets you assess forecasted sales at world market, submarket and national level. You will see financial results, interviews, trends, opportunities and revenue predictions.

Forecasts from 2015-2025 and other analyses show you commercial prospects Besides revenue forecasting to 2025, our new study provides you with recent results, growth rates, and market shares. There you will find original analyses, with business outlooks and developments. Discover qualitative analyses (including SWOT and Porter's Five Forces), company profiles and commercial developments. Read the full transcript of an exclusive expert opinion interview from industry specialists informing your understanding and allowing you to assess prospects for investments and sales: Dr Antonio SJ Lee, CEO and Managing Director, MEDIPOST America Inc.

You find prospects for key submarkets and products In addition to analyses of the overall world market, you see revenue forecasts for these three submarkets to 2025: Stem cell therapies Gene Therapies Tissue engineering products

Products that can significantly increase disease-free survival and improve patient tolerance will achieve success. In the long term, we forecast these curative therapies to be adopted by many healthcare systems globally.

Our investigation shows business research and analyses with individual revenue forecasts and discussions. You find dynamics of the industry and assess its potential sales, seeing agents likely to achieve the most success.

To see a report overview please email Sara Peerun on sara.peerun@visiongainglobal.com

See revenue forecasts for products How will leading products perform to 2025 at the world level? Our study forecasts sales of currently marketed and pipeline regenerative medicine products including these: Osteocel Plus Trinity ELITE and Trinity Evolution Prochymal Apligraf Dermagraft ReCell Neovasculgen Glybera Talimogene Laherparepvec (T-Vec)

Discover how high revenues can go. You will see what is happening, understanding trends, challenges and opportunities.

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Translational Regenerative Medicine: Market Prospects 2015-2025

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Durham, NC (PRWEB) February 27, 2015

Several studies showing the promise of stem cells for treating patients with heart failure have made headline news recently. However, all these studies dealt with adult patients only. New research appearing in this months STEM CELLS Translational Medicine shows that stem cells may have the same potential in treating children with congenital heart diseases that can lead to heart failure.

The study, undertaken by researchers at the Mayo Clinic in Rochester, Minn., looked at the feasibility and long-term safety of injecting autologous umbilical cord blood cells directly into the heart muscle at the pediatric stage of heart development. The study was conducted on pigs, due to their hearts similarity to human hearts.

The team injected the stem cells directly into the right ventricle of groups of three- and four-week old healthy piglets, and then compared the results to a control group that did not receive any cells. Over the next three months, the animals were monitored to assess cardiac performance and rhythm to determine how safe the procedure would be for humans.

During this follow-up period, we found no significant acute or chronic cardiac injury pattern caused by the injections directly into the heart, said lead author Timothy J. Nelson, M.D., Ph.D., of the Mayo Clinics Department of Medicine, and all the animals hearts appeared to be normal and healthy.

This led us to conclude that autologous stem cells from cord blood can be safely collected and surgically delivered to children. The study also establishes the foundation for cell-based therapy for children and aims to accelerate the science toward clinical trials for helping children with congenital heart disease that could benefit from a regenerative medicine strategy, he added.

The lead author, Susan Cantero Peral, M.D., Ph.D. commented, This work highlights the importance and utility of umbilical cord blood as it can be applied to new applications. Rather than discarding this sample at birth, individuals with congenital heart disease may one day be able to have these cells collected and processed in a specialized way to make them available for cardiac regeneration.

This work was funded by the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome founded at the Mayo Clinic.

These data help establish the foundation of a cell-based therapy for juvenile hearts by showing that injections of autologous cells from cord blood are safe and feasible, said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

###

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Study Shows Stem Cells Have Potential to Help Kids Hearts, Too

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Albany, NewYork (PRWEB) February 27, 2015

ResearchMoz has announced the addition of a recent study that presents the analysis of the cell culture protein surface coating market across the globe. The research report discusses the current scenario and development prospects of the global cell culture protein surface coating industry for the period of 2015 to 2019.

Read Complete Report With TOC @ http://www.researchmoz.us/global-cell-culture-protein-surface-coating-market-2015-2019-report.html

The research report, titled Global Cell Culture Protein Surface Coating Market, offers an analytical study, providing an in-depth assessment of the industry based on market trends, growth drivers as well as challenges. This is done taking various segments of the market into consideration. The report also forecasts that the worldwide cell culture protein surface coating industry will expand at a CAGR of 12.91% during the forecast period of 2014 to 2019.

Cell culture protein surface coating is defined as the coating process wherein cell culture surfaces are covered with extra-cellular matrix elements or with protein to improve in-vitro linkage and propagation in the cells.

The various kinds of proteins that are available in our surroundings are synthetic proteins, human-derived proteins, plant-derived proteins, and animal-derived proteins. Fibronectin, collagen, laminin, osteopontin, and vitronectin are some of the proteins that are utilized for cell culture protein surface coating. Cell culture protein surface coating assists in the development of several kinds of cells such as epithelial, endothelial, fibroblasts, muscle cells and myoblasts, leukocytes, CHO cell lines, and neurons.

The wide range of applications for cell culture protein surface coatings consist of enhanced adhesion of cells, better propagation and development of cells, cell matrix studies, morphogenesis studies, receptor-ligand binding studies, signal transduction studies, genetic engineering, differentiation of individual cell types, drug screening, and metabolic pathway studies.

Stem cells have high potential for the treatment of severe diseases such as cardiac ailments, neuro degenerative diseases, and even diabetes. This fact has resulted in the increase in demand for highly developed cell culture products for stem cell manufacturing and studies. Cell culture protein surface coating offers enhanced adhesion, propagation, and rapid development of cells during the period of isolation and cultivation.

The main factor that is adding to the growth of the global cell culture protein surface coating industry is increased focus of top market players towards stem cell research. However, the drawbacks of animal-derived protein surface coating is a factor that is soon becoming a matter of concern, hindering the growth of the cell culture protein surface coating market.

Top players of the cell culture protein surface coating industry are EMD Millipore, Thermo Fisher Scientific, Becton, Dickinson and Company, Corning, and Sigma-Aldrich.

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Global Cell Culture Protein Surface Coating Industry: Rising Focus towards Stem Cells Research to Trigger Market Growth

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Since the first experimental bone marrow transplant over 50 years ago, more than one million hematopoietic stem cell transplantations (HSCT) have been performed in 75 countries, according to new research charting the remarkable growth in the worldwide use of HSCT, published in The Lancet Haematology journal.

However, the findings reveal striking variations between countries and regions in the use of this lifesaving procedure and high unmet need due to a chronic shortage of resources and donors that is putting lives at risk.

HSCT (also known as blood and marrow transplant) is most often used to treat diseases of the blood and several types of cancer such as multiple myeloma or leukaemia. For many people with these diseases the only possibility of a cure is to have a HSCT. The procedure provides healthy cells from either the patient (autologous transplantation) or from a healthy donor (allogeneic transplantation) to replace those lost to disease or chemotherapy.

Using data collected by the Worldwide Network for Blood and Marrow Transplantation (WBMT), Professor Dietger Niederwieser from the University Hospital Leipzig in Germany and international colleagues, systematically analysed the growth of HSCT and changes in its use in 194 WHO member countries since the first transplant in 1957. They also examined the link between macroeconomic factors (eg, gross national income and health care expenditure) and transplant frequencies per 10 million inhabitants in each country.

Although only a small number of centres had performed about 10000 transplants by 1985, this had risen to around 500000 ten years later, and doubled to more than 1 million transplants (42% allogeneic and 58% autologous) done at 1516 transplant centres across 75 countries by the end of December 2012 (see table 1 page 2).

Perhaps unsurprisingly, the study found that transplants are more common in countries with greater financial resources and more institutions with the resources and expertise to perform HSCT. Most of the HSCTs have been performed in Europe (53%), followed by the Americas (31%), South East Asia and Western Pacific (15%), and the Eastern Mediterranean and Africa (2%). The findings also reveal significant differences between HSCT use by donor type (autologous or allogeneic), indications for HSCT, and world region (See tables 2, 3, and 4 pages 4-6). For example, donor transplants in 2010 ranged in active countries from 0.4 per 10 million inhabitants in the Philippines and Vietnam to 506 in Israel (see figure 2B page 7).

Numbers of donor transplants have rapidly expanded in all regions without any signs of saturation (see table 1 page 2). This is likely to reflect substantial underuse of this therapy, say the authors, suggesting that more patients would have been treated with allogeneic transplantation had it been accessible, or had suitable donors been available.

In about 30% of cases, a genetically matched donor can be found from within a patient's family. The other 70% have to search for a matched volunteer from national and international registries. The report shows that numbers of countries with registries increased from 2 in 1987 to 57 in 2012, whilst volunteer donors rose from 3072 in 1987 to over 22 million in 2012. The international exchange of stem-cell products also increased to more than 10000 a year between 2006 and 2012, with substantial differences between countries in the amount of stem cells they import or export (see figure 2C page 7).

Despite these increases there are still too many patients who are unable to find a suitable donor. At any time around 1800 people in the UK are waiting for a blood stem cell donation, and over 37000 people are waiting worldwide. Moreover, less than half of the people in the UK diagnosed with a blood cancer ever find a suitable donor [1].

According to Professor Niederwieser, "Patients, many of them children, are facing a life and death situation. Ultimately they will die if they cannot get the treatment they need. All countries need to provide adequate infrastructure for patients and donors to make sure that everyone who needs a transplant gets one, rather than the present situation in which access remains restricted to countries and people with sufficient resources."[2]

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The Lancet Haematology: Experts warn of stem cell underuse

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Riverside, ON and Brea CA (PRWEB) February 26, 2015

The Irvine Stem Cell Treatment Center announces a series of free public seminars on the use of adult stem cells for various degenerative and inflammatory conditions. They will be provided by Dr. Thomas A. Gionis, Surgeon-in-Chief.

The seminars will be held on Saturday, March 7, 2015, at 11:00 am, 1:00 pm and 3:00 pm at Courtyard Riverside Downtown / Marriott, 1510 University Avenue, Riverside, CA 92507; Tuesday, March 10, 2015, at 11:00 am, 1:00 pm and 3:00 pm at Ayres Suites Ontario at the Mills Mall, 4370 Mills Circle, Ontario, CA 91764; and Saturday, March 21, 2015, at 11:00 am, 1:00 pm and 3:00 pm at Embassy Suites Hotel, 900 E Birch Street, Brea, CA 92821. Please RSVP at (949) 679-3889.

The Irvine Stem Cell Treatment Center (Irvine and Westlake), along with sister affiliates, the Miami Stem Cell Treatment Center (Miami; Boca Raton; Orlando; The Villages, Florida) and the Manhattan Regenerative Medicine Medical Group (Manhattan, New York), abide by approved investigational protocols using adult adipose derived stem cells (ADSCs) which can be deployed to improve patients quality of life for a number of chronic, degenerative and inflammatory conditions and diseases. ADSCs are taken from the patients own adipose (fat) tissue (found within a cellular mixture called stromal vascular fraction (SVF)). ADSCs are exceptionally abundant in adipose tissue. The adipose tissue is obtained from the patient during a 15 minute mini-liposuction performed under local anesthesia in the doctors office. SVF is a protein-rich solution containing mononuclear cell lines (predominantly adult autologous mesenchymal stem cells), macrophage cells, endothelial cells, red blood cells, and important Growth Factors that facilitate the stem cell process and promote their activity.

ADSCs are the bodys natural healing cells - they are recruited by chemical signals emitted by damaged tissues to repair and regenerate the bodys injured cells. The Irvine Stem Cell Treatment Center only uses Adult Autologous Stem Cells from a persons own fat No embryonic stem cells are used; and No bone marrow stem cells are used. Current areas of study include: Emphysema, COPD, Asthma, Heart Failure, Heart Attack, Parkinsons Disease, Stroke, Traumatic Brain Injury, Lou Gehrigs Disease, Multiple Sclerosis, Lupus, Rheumatoid Arthritis, Crohns Disease, Muscular Dystrophy, Inflammatory Myopathies, and degenerative orthopedic joint conditions (Knee, Shoulder, Hip, Spine). For more information, or if someone thinks they may be a candidate for one of the adult stem cell protocols offered by the Irvine Stem Cell Treatment Center, they may contact Dr. Gionis directly at (949) 679-3889, or see a complete list of the Centers study areas at: http://www.IrvineStemCellsUSA.com.

About the Irvine Stem Cell Treatment Center: The Irvine Stem Cell Treatment Center, along with sister affiliates, the Miami Stem Cell Treatment Center and the Manhattan Regenerative Medicine Medical Group, is an affiliate of the California Stem Cell Treatment Center / Cell Surgical Network (CSN); we are located in Irvine and Westlake, California. We provide care for people suffering from diseases that may be alleviated by access to adult stem cell based regenerative treatment. We utilize a fat transfer surgical technology to isolate and implant the patients own stem cells from a small quantity of fat harvested by a mini-liposuction on the same day. The investigational protocols utilized by the Irvine Stem Cell Treatment Center have been reviewed and approved by an IRB (Institutional Review Board) which is registered with the U.S. Department of Health, Office of Human Research Protection (OHRP); and our studies are registered with Clinicaltrials.gov, a service of the U.S. National Institutes of Health (NIH). For more information, visit our websites: http://www.IrvineStemCellsUSA.com, http://www.MiamiStemCellsUSA.com, or http://www.NYStemCellsUSA.com.

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A team of European researchers has devised a strategy to ensure that adult epidermal stem cells are safe before they are used as treatments for patients. The approach involves a clonal strategy where stem cells are collected and cultivated, genetically modified and single cells isolated before being rigorously tested to make sure they meet the highest possible safety criteria. The strategy, which is published online in EMBO Molecular Medicine, is inspired by the approaches the biotechnology industry and regulatory affairs authorities have adopted for medicinal proteins produced from genetically engineered mammalian cells.

"Until now there has not been a systematic way to ensure that adult epidermal stem cells meet all the necessary requirements for safety before use as treatments for disease," says EMBO Member Yann Barrandon, Professor at Lausanne University Hospital, the Swiss Federal Institute of Technology in Lausanne and the lead author of the study. "We have devised a single cell strategy that is sufficiently scalable to assess the viability and safety of adult epidermal stem cells using an array of cell and molecular assays before the cells are used directly for the treatment of patients. We have used this strategy in a proof-of-concept study that involves treatment of a patient suffering from recessive dystrophic epidermolysis bullosa, a hereditary condition defined by the absence of type VII collagen which leads to severe blistering of the skin."

The researchers cultivated epidermal cells from the patient that can be used to regenerate skin. The scientists used their array of tests to determine which of the transduced cells met the necessary requirements for stemness -- the characteristics of a stem cell that distinguish it from a regular cells -- and safety. Clonal analysis revealed that the transduced stem cells varied in their ability to produce functional type VII collagen. When the most viable, modified stem cells were selected, transplantation onto immunodeficient mice regenerated skin that did not blister in the mouse model system for recessive dystrophic epidermolysis bullosa and produced functional type VII collagen. Safety was assessed by determining the sites of integration of the viral vector, looking for rearrangements and hit genes, as well as whole genome sequencing.

"Our work shows that at least for adult epidermal stem cells it is possible to use a clonal strategy to deliver a level of safety that cannot be obtained by other gene therapy approaches. A clonal strategy should make it possible to integrate some of the more recent technologies for targeted genome editing that offer more precise ways to change genes in ways that may further benefit the treatment of disease. Further work is in progress in this direction."

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The above story is based on materials provided by EMBO - excellence in life sciences. Note: Materials may be edited for content and length.

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Quality control for adult stem cell treatment

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Newswise In a study published today (February 26) in Cell, researchers at Rockefeller University home in on one culprit that fuels this variable vulnerability within squamous cell cancers: exposure to a signal known as TGF-, given off by immune cells that congregate next to a tumors blood vessels.

There are several reasons why some cancer stem cells, the cells at the root of tumors and metastases, can withstand therapy meant to eradicate them. Our results point to the importance of the environment immediately surrounding the skin cancer stem cells, specifically, their exposure to the signal TGF-, says senior researcher Elaine Fuchs, Rebecca C. Lancefield Professor, head of the http://Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Developmenthttp://lab.rockefeller.edu/fuchs/ and a Howard Hughes Medical Institute Investigator. Ultimately, we hope this new insight could lead to better means for preventing the recurrence of these life-threatening cancers, which can occur in the skin, head, neck, esophagus, and lung, and often evade treatment.

Her team, which included first author Naoki Oshimori, a postdoctoral research associate in the lab and lab technician Daniel Oristian, focused on squamous cell carcinomas in the skin of mice. Like many normal tissue stem cells, the stem cells that produce squamous cell tumors can be classified into two types: those that divide and proliferate rapidly, and those that do so more slowly. This has led scientists to wonder whether the more dormant stem cells in a tumor might evade cancer drugs.

To investigate this possibility, the team zeroed in on TGF- (transforming growth factor beta) which is known to restrict growth in many healthy tissues. The labs previous research has shown that mice whose normal skin stem cells cannot respond to TGF- become susceptible to develop tumors that grow rapidly. Paradoxically, however, TGF- contributes to metastasis in many cancers. The researchers wanted to know: How can TGF- act both to suppress cancers and promote them?

By visualizing TGF- signaling within developing mouse tumors, the researchers found that the cancer stem cells located nearest to the blood vessels of the tumor receive a strong TGF- signal, while others further away dont receive any. To see this difference and its effects, they used a red tag to illuminate those cells exposed and responding to TGF-, and a green genetic tag, which they could switch on, to track the stem cells progeny. Over time, they saw that TGF--responding stem cells proliferate more slowly but they simultaneously invade, scatter and move away from the tumor. The opposite was true of cancer stem cells too far away to receive TGF-, which proliferated rapidly, but were less invasive, growing as a tumor mass.

We tested the implications for drug resistance by injecting cisplatin, a commonly used chemotherapy drug for these types of cancers, into the mice with tumors. While the drug killed off most of the TGF- nonresponding cancer cells, it left behind many of the responders, Oshimori says. When the drug was withdrawn, the lingering TGF- responding cancer stem cells grew back the tumor.

We found that the TGF- heterogeneity in the tumor microenvironment produces some cancers stem cells that divide rapidly and lead to accelerated tumor growth, and other cancer stem cells that invade surrounding healthy tissue and escape cancer therapies, Fuchs explains. Moreover, conventional wisdom might say that a leisurely pace of cell division, like that seen in the TGF- responders, makes it possible for these cells to circumvent anticancer treatments that target rapidly dividing cells. While this may be true for some types of anticancer drugs, we found changes in antioxidant activity in these cells are more important for their resistance to cisplatin.

Indeed, when the team compared the genes expressed by the TGF- responders with those of the nonresponders, they found highly elevated expression in a battery of genes encoding enzymes involved in making and utilizing glutathione, an important antioxidant and detoxifying substance in cells. This unexpected finding led the team to test the impact of glutathione metabolism and conclude this metabolic pathway prevents TGF- responders from critical damage by anti-cancer drugs as well as oxidative stresses.

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Growth Signal Can Influence Cancer Cells' Vulnerability to Drugs, Study Suggests

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Newswise NEW YORK, NY (February 27, 2015) Researchers have for the first time successfully converted adult human skin cells into neurons of the type that regulate appetite, providing a patient-specific model for studying the neurophysiology of weight control and testing new therapies for obesity. The study, led by researchers at Columbia University Medical Center (CUMC) and at the New York Stem Cell Foundation (NYSCF), was published last month in the online issue of the Journal of Clinical Investigation.

In a separate study, which appeared in the February 10 issue of the journal Development, Kevin Eggan, PhD, Florian Merkle, and Alexander Schier of Harvard University have also succeeded in creating hypothalamic neurons from iPS cells. These neurons help to regulate behavioral and basic physiological functions in the human body, including, in addition to appetite, hypertension, sleep, mood, and some social disorders. The investigators at Columbia and Harvard shared ideas during the course of the research, and these studies are co-validating.

Mice are a good model for studying obesity in humans, but it would better to have human cells for testing. Unfortunately, the cells that regulate appetite are located in an inaccessible part of the brain, the hypothalamus. So, until now, weve had to make do with a mouse model or with human cells harvested at autopsy. This has greatly limited our ability to study fundamental aspects of human obesity, said senior author Rudolph L. Leibel, MD, the Christopher J. Murphy Memorial Professor of Diabetes Research, professor of pediatrics and medicine, and co-director of the Naomi Berrie Diabetes Center at CUMC.

To make the neurons, human skin cells were first genetically reprogrammed to become induced pluripotent stem (iPS) cells. Like natural stem cells, iPS cells are capable of developing into any kind of adult cell when given a specific set of molecular signals in a specific order. The iPS cell technology has been used to create a variety of adult human cell types, including insulin-producing beta cells and forebrain and motor neurons. But until now, no one has been able to figure out how to convert human iPS cells into hypothalamic neurons, said co-author Dieter Egli, PhD, assistant professor of pediatrics (in developmental cell biology), a member of the Naomi Berrie Diabetes Center, and a senior research fellow at NYSCF.

This is a wonderful example of several institutions coming together to collaborate and advance research in pursuit of new therapeutic interventions. The ability to make this type of neuron brings us one step closer to the development of new treatments for obesity, said Susan L. Solomon, CEO of NYSCF.

The CUMC/NYSCF team determined which signals are needed to transform iPS cells into arcuate hypothalamic neurons, a neuron subtype that regulates appetite. The transformation process took about 30 days. The neurons were found to display key functional properties of mouse arcuate hypothalamic neurons, including the ability to accurately process and secrete specific neuropeptides and to respond to metabolic signals such as insulin and leptin.

We dont think that these neurons are identical to natural hypothalamic neurons, but they are close and will still be useful for studying the neurophysiology of weight control, as well as molecular abnormalities that lead to obesity, said Dr. Leibel. In addition, the cells will allow us to evaluate potential obesity drugs in a way never before possible.

This shows, said Dr. Eggan, how improved understanding of stem cell biology is making an impact on our ability to study, understand, and eventually treat disorders of the nervous system. Because there are so few hypothalamic neurons of a given type, they have been notoriously difficult to study. The successful work by both groups shows that this problem has been cracked.

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Neurons Controlling Appetite Made From Skin Cells

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New York City--A novel phase 1 clinical trial that leverages T-cell immunotherapy is now under way at Karmanos Cancer Institute (KCI) in Detroit and Memorial Sloan-Kettering Cancer Center in New York City; bringing new hope to children and young adults with osteosarcoma and neuroblastoma. This new clinical trial is being funded by charity partners Solving Kids' Cancer and Fishin' For The Cure.

T-cell therapy to treat both adult and childhood cancers has been in recent news for the dramatic responses seen in some patients, who've experienced a complete remission or have no more cancer cells in their body. A type of immunotherapy, T-cell therapy trains a patient's own white blood cells that are collected and engineered to recognize specific targets on the surface of cancer cells. These "armed" T-cells are cloaked with a bi-specific T-cell engaging antibody (BiTE) using a new humanized antibody called hu3F8, and returned to the patients as a vaccine with one mission, kill cancer cells. Scientists believe that by incorporating this BiTE antibody, the T-cells will be more effective.

"Children and young adults battling osteosarcoma and neuroblastoma need curative treatment options after chemotherapy and targeted drugs stop working," said Tracy Russo, the Executive Director of Fishin' For The Cure. "We are excited about the possibilities."

This new T-cell therapy targets GD2, an antigen on the tumor cell surface in over 90 percent of osteosarcomas and neuroblastoma and CD3, a receptor found on healthy T-cells. This dual-targeted approach using CD3 and another target has been successfully shown to be safe and elicited immune resonses in breast cancer patients. In addition, the armed T-cells train the other T-cells in the body to continue to recognize and kill tumor cells.

"As patients receive multiple infusions of this new T-cell therapy, which in essence 'vaccinates' patients against their own cancer cells, it has the potential to induce long-term immune responses while being less toxic," said Scott Kennedy, the Executive Director of Solving Kids' Cancer.

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A novel immunotherapy technique to treat patients with osteosarcoma and neuroblastoma

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