Archive for the ‘Gene Therapy Research’ Category

Details Published on Friday, 27 July 2012 00:00

STEM cell therapy is being eyed to cure the neck and back pains and other illnesses of former president and now Pampanga Rep. Gloria Arroyo, alternative medicine doctor Antonia Park said yesterday.

Arroyo went to Parks Green and Young Health and Wellness Center in Tagaytay City yesterday morning for cleansing and alternative healing.

A guard at the La Vista Subdivision in Quezon City, who requested anonymity, said Arroyo left the subdivision at around 7:30 a.m., accompanied by a few staff and a personal nurse. She rode a white Nissan Patrol and her convoy included a gray Toyota Land Cruiser and a police escort.

Arroyo was granted bail Wednesday by a Pasay City court after finding that the electoral sabotage case against her was weak. She posted a P1-million cash bond.

Stem cell treatment involves the introduction of new adult stem cells into the damaged tissue in order to treat a disease or injury. The ability of new cells to regenerate is seen as having significant potential to replace diseased areas of the body, with minimal risk of rejection and side effects.

Park, in a statement distributed to the media, said Arroyo complained of difficulty in swallowing with choking due to her bulge along the posterior pharyngeal wall, together with a change of her voice and losing weight due to her difficulties of swallowing the food (solid) and angina as well as continuous neck and back pain.

Thats why our center is accepting her for possible stem cell therapy and another treatment of pain management and giving natural food by means of fresh fruit and vegetable juices for which management is warranted and which the stem cell therapy is contemplated and strongly considered, she said.

She said the stem cell therapy can be given by her clinic in Tagaytay while Arroyo can continue her physical therapy at the Veterans Memorial Medical Center four times a week.

Park said Arroyo can to go her district, referring to the second district of Pampanga, over the weekend provided she takes care to wear a brace and avoid talking too much so as to protect the bulging interior of the throat, and provided she resumes physical therapy as soon as possible.

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Malaya Business Insight

MANILA, Philippines - Former President Gloria Macapagal-Arroyo might undergo stem cell therapy to improve her health, according to the alternative medicine facility in Tagaytay City that the Pampanga lawmaker visited Thursday.

Arroyo came to the facility complaining of difficulty in swallowing because of a bulge in her throat, according to a statement from the Green 8 Young Health & Wellness Center.

Her voice has also changed and she is losing weight because she can't swallow solid food. She also has angina, the center said.

Arroyo also complained of continuing neck and back pain.

"Our center is accepting her for possible stem cell therapy," the alternative medicine facility said. "The stem cell therapy is... strongly considered."

It said the Arroyo can undergo such therapy in the Tagaytay center while her physical therapy will continue at the Veterans Memorial Medical Center 4 times a week.

The Pampanga lawmaker is seeking treatment at the center through her sister, Cielo Macapagal-Salgado.

The center said Salgado was previously diagnosed with a cancerous lump in her breast.

"She consulted several doctors and was subsequently subjected to a myrad of treatment procedures. These, however, did not produce the desired results. When she came to our center she was cured of her cancer," it claimed.

Arroyo heads to Pampanga

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Arroyo might undergo stem cell therapy

Public release date: 26-Jul-2012 [ | E-mail | Share ]

Contact: Steve Graff stephen.graff@jefferson.edu 215-955-5291 Thomas Jefferson University

PHILADELPHIABreast cancer patients whose tumors lacked the retinoblastoma tumor suppressor gene (RB) had an improved pathological response to neoadjuvant chemotherapy, researchers at Thomas Jefferson University Hospital and the Kimmel Cancer Center at Jefferson report in a retrospective study published in a recent online issue of Clinical Cancer Research.

Many breast cancer patients undergo neoadjuvant therapy to reduce the size or extent of the cancer before surgical intervention. Complete response of the tumor to such treatment signifies an improved overall prognosis. Today, no marker is applied to identify tumors which will respond to such treatment, and as a result, only a subset of patients exhibit benefit from it.

"We found that loss of RB was associated with better pathological response rates in breast cancer patientsat various stages and representing multiple molecular subtypeswho were administered neoadjuvant chemotherapy," said Agnieszka Witkiewicz, M.D., Associate Professor of Pathology, Anatomy and Cell Biology at Thomas Jefferson University.

Erik Knudsen, Ph.D, Professor of Cancer Biology and the Hilary Koprowski Chair in Cancer Biology, was excited that discoveries from his life-long research on the RB-pathway were making there way into the clinic.

"This represents a potential new biomarker that could be used to tailor treatment plans for women considering neoadjuvant therapy and is a testament to the importance of cancer research," he said.

For the study, researchers, including Gordon Schwartz, M.D., Director of the Jefferson Breast Care Center and Adam Ertel, Ph.D., Bioinformatics Specialist, Department of Cancer Biology, performed a combination of gene expression profiling to identify those with RB loss and direct histological analysis in over 1,000 breast cancer patients who had undergone neoadjuvant therapy. These patients represented distinct subtypes of breast cancer and were treated with multiple different therapeutic regimens.

RB loss was associated, the team found, with an improved response to all the neoadjuvant regimens investigated in the major subtypes of breast cancer.

"Together, these data indicate that the loss of RB, which occurs relatively frequently in locally advanced disease, could be a useful tool for defining patients that experience an improved response to neoadjuvant chemotherapy," said Dr. Witkiewicz. "Based on these findings, we have initiated a prospective clinical trial at Jefferson, evaluating the association of RB and another marker, PTEN, with the response to neoadjuvant chemotherapy."

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Breast cancer patients who lack RB gene respond better to neoadjuvant chemotherapy

Public release date: 26-Jul-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 26, 2012 Administering high-doses of interleukin-2 (IL-2) has been the preferred treatment for patients with stage IV metastatic melanoma. An article published in the current issue of Cancer Biotherapy and Radiopharmaceuticals, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com), explores whether or not this regimen is still the most effective. The article is available free online at the Cancer Biotherapy and Radiopharmaceuticals website (http://www.liebertpub.com/cbr).

In the article "Should High-Dose Interleukin-2 Still Be the Preferred Treatment for Patients with Metastatic Melanoma?" (http://online.liebertpub.com/doi/full/10.1089/cbr.2012.1220) Robert Dillman and colleagues at the Hoag Institute for Research and Education and Hoag Family Cancer Institute, Newport Beach, CA concluded that until long-term survival data for some of the newer drugs are available, patients with stage IV metastatic melanoma who are well enough to be given intensive IL-2 therapy should receive it initially, either alone or in combination with one of the newer therapeutic agents.

"This is an important article that puts into perspective the reasons why IL-2 should continue to be the initial therapy in patients with metastatic melanoma," says Editor Donald J. Buchsbaum, PhD, Division of Radiation Biology, Department of Radiation Oncology, University of Alabama at Birmingham.

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About the Journal

Cancer Biotherapy and Radiopharmaceuticals (http://www.liebertpub.com/cbr), published 10 times a year in print and online, is under the editorial leadership of Editors Donald J. Buchsbaum, PhD and Robert K. Oldham, MD, Lower Keys Cancer Center, Key West, FL. Cancer Biotherapy and Radiopharmaceuticals is the only journal with a specific focus on cancer biotherapy, including monoclonal antibodies, cytokine therapy, cancer gene therapy, cell-based therapies, and other forms of immunotherapy. The Journal includes extensive reporting on advancements in radioimmunotherapy and the use of radiopharmaceuticals and radiolabeled peptides for the development of new cancer treatments. Topics include antibody drug conjugates, fusion toxins and immunotoxins, nanoparticle therapy, vascular therapy, and inhibitors of proliferation signaling pathways.

About the Publisher

Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Journal of Interferon & Cytokine Research, Human Gene Therapy and Human Gene Therapy Methods, and Stem Cells and Development. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available at Mary Ann Liebert, Inc. (http://www.liebertpub.com)

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Should high-dose interleukin-2 continue to be the treatment of choice for metastatic melanoma?

SAN DIEGO, July 26, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today reported revenue of $18.3 million for the second quarter of 2012, an increase of 23% from revenue of $14.9 million in the first quarter of 2012 and a 38% increase compared to revenue of $13.3 million for the second quarter of 2011. Second quarter 2012 revenues from the Sequenom Center for Molecular Medicine (Sequenom CMM) diagnostic services operating segment grew more than 400%, while revenues from the genetic analysis operating segment decreased 14% in the second quarter of 2012 as compared to the prior year period.

"We have continued to see strong increases in the uptake of the MaterniT21 PLUS laboratory developed test (LDT) and Sequenom CMM has now analyzed more than 20,000 test samples since the product was launched in October of last year. We have been able to effectively meet this increasing demand by expanding our operational capacity so that we can continue to meet the needs of our physician customers and their patients in a prompt and timely manner," said Harry Hixson, Jr., Ph.D., Chairman and CEO of Sequenom. "We believe that our operational model, additional efforts and test enhancements planned for the second half of the year will position us well for sustainable growth and product leadership going forward."

Gross margin in the second quarter of 2012 was 32% of revenues as compared to a gross margin of 67% in the same period one year ago, a difference primarily attributable to the cost associated with the launch and increasing market adoption of the Sequenom CMM MaterniT21 PLUS LDT. Diagnostic services revenue is primarily recognized when cash is received, while costs are recognized in the current period upon completion of the services. The costs associated with increasing test volumes resulted in decreased gross margins as the Company has not yet recognized revenue related to the increased number of tests performed, which have been billed but not yet collected. Gross margin is expected to continue to fluctuate quarterly due to changes in sales volumes and the timing of cash receipts until the Company converts to accrual accounting for diagnostic services revenue, which is expected to occur after sufficient reimbursement history has been established.

Total operating expenses for the second quarter of 2012 were $35.1 million, as compared to total expenses of $29.9 million for the second quarter of 2011. This change reflects increased selling and marketing expenses resulting primarily from higher labor costs associated with increased headcount to support operations and the continued expansion of the diagnostic services infrastructure. Research and development expenses decreased 19% from the prior year period, primarily due to a reduction in research-related licensing and collaboration costs.

General and administrative expenses increased in the second quarter of 2012 to $9.9 million, an increase due primarily to facilities and legal expenses, and higher labor costs associated with increased headcount to support operations as compared to the same period one year ago. Total stock-based compensation expense was $3.2 million for the second quarter of 2012, as compared to $3.5 million recorded during the second quarter of 2011.

Net loss for the second quarter of 2012 was $29.6 million, or $0.26 per share, as compared to net loss of $20.9 million, or $0.21 per share for the same period in 2011, resulting from an increase in costs associated primarily with the growth in testing volume of the MaterniT21 PLUS LDT.

"Even as we continue to invest in strategies to expand the reach of Sequenom CMM's testing services, our second quarter results are generally in line with our expectations given the rapid adoption of the MaterniT21 PLUS LDT," said Paul V. Maier, Sequenom's CFO. "Importantly, for the first time, this quarter the MaterniT21 PLUS LDT made the largest contribution to Sequenom's diagnostic services revenue, a trend we expect to continue moving forward. We expect our diagnostic services margins to improve in the second half of 2012 as Sequenom CMM implements further test enhancements and the steps to achieve the benefits of volume growth."

First Half Results For the first half of 2012, the Company reported revenue of $33.2 million, an increase of 24% from revenue of $26.8 million for the first half of 2011. Revenues in the first half of 2012 from the Sequenom CMM diagnostics services operating segment grew 295%, while revenues from the genetic analysis operating segment decreased 14% in the first half of 2012 as compared to the prior year period.

Gross margin for the first half of 2012 was 34% of revenue as compared to gross margin of 64% for the first half of 2011, a difference primarily attributable to the cost associated with the increased test volume resulting from the launch and increased market adoption of the Sequenom CMM MaterniT21 PLUS LDT.

Total operating expenses for the first half of 2012 were $64.9 million, as compared to total expenses of $51.4 million for the first half of 2011. This change reflects increased selling and marketing expenses resulting primarily from higher labor costs associated with increased headcount to support operations and the continued expansion of the diagnostic services infrastructure. Research and development expenses decreased 4% as compared to the first half of 2011, primarily due to a reduction in research-related licensing and collaboration costs.

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Sequenom, Inc. Reports Financial Results For The Second Quarter Of 2012 And Increases Full-Year MaterniT21™ PLUS Test ...

Western Pygmies

I love population genetics for its ability to peer back into human history through the medium of DNAs ATCGs.

One of the stars of this discipline is Sarah Tishkoff, a standout in African genetics, someone who will readily haul a centrifuge into the bush in Cameroon.

Tishkoff of the University of Pennsylvania is lead author on a paper published online July 26 inCell that details whole-genome sequencing of five individuals each from three extant hunter-gatherer groupsthe Pygmies of Cameroon as well as the Hadza and the Sandawe of Tanzania. The results reveal millions of newly discovered genetic variantsdifferences in single genetic letters, the ATCGsand indicate that early modern humans may have interbred long ago in Africa with another species of hominid (although the fossil record does not provide much support for the latter finding).

Tishkoff answered a few questions for us about this paper, co-authored with Joseph Lachance and 11 other researchers. An edited version of the interview appears below:

Please describe the research that led to the paper that was published today:

Were the first ones to look at these diverse groups of hunter-gathers in Africa who descend from some of the most ancestral lineages in the world. Theyre interesting because they have very unique and distinct lifestyles There are few populations that maintain this active hunter-gatherer lifestyle.

This is the most extensive study in Africa using high-coverage deeply detailed sequence data. We focused on three groups because theyre anthropologically interesting. Theyre thought to be descended from groups that are ancestral to all modern humans. We wanted to understand the genetic basis of adaptation to their local environment including, for instance the short stature trait in Pygmies.

So what did you find?

We discovered 13 million variants and, of those variants, greater than 3 million are completely novel, meaning that they have not been reported in any database. The current public database has 40 million variants. So we found 3 million novel variants by simply sequencing 15 individuals. That increases by about 8 percent all known human genetic variation. It also demonstrates that were missing a lot of really important variation thats out there, particularly in Africa, which is the homeland of modern humans and a place where theres been a lot of time for differentiation to have occurred in very diverse environments. What this means is that theres s probably a lot of regional or population-specific variation out there that has not been that well characterized, some of which is functionally very important.

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Out of Africa: Startling New Genetics of Human Origins

BOTHELL, Wash.--(BUSINESS WIRE)--

Seattle Genetics, Inc. (SGEN) announced today that it will report its second quarter and year-to-date 2012 financial results on Wednesday, August 8, 2012, after the close of financial markets. Following the announcement, company management will host a conference call and webcast discussion of the results and provide a general corporate update. Access to the event can be obtained as follows:

LIVE access on Wednesday, August 8, 2012

1:30 p.m. Pacific Time (PT) / 4:30 p.m. Eastern Time (ET)

REPLAY access

About Seattle Genetics

Seattle Genetics is a biotechnology company focused on the development and commercialization of monoclonal antibody-based therapies for the treatment of cancer. The U.S. Food and Drug Administration granted accelerated approval of ADCETRIS in August 2011 for two indications. ADCETRIS is being developed in collaboration with Millennium: The Takeda Oncology Company. In addition, Seattle Genetics has three other clinical-stage antibody-drug conjugate (ADC) programs: SGN-75, ASG-5ME and ASG-22ME. Seattle Genetics has collaborations for its ADC technology with a number of leading biotechnology and pharmaceutical companies, including Abbott, Bayer, Celldex Therapeutics, Daiichi Sankyo, Genentech, GlaxoSmithKline, Millennium, Pfizer and Progenics, as well as ADC co-development agreements with Agensys, an affiliate of Astellas, and Genmab. More information can be found at http://www.seattlegenetics.com.

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Seattle Genetics to Host Conference Call and Webcast Discussion of Second Quarter 2012 Financial Results on August 8 ...

SALT LAKE CITY, July 26, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced that it has signed an agreement with Pharma Mar, a leader in the development of marine-derived drugs. Under the agreement, Myriad will conduct BRCA 1 and BRCA 2 testing on patients to be enrolled in a Phase II clinical study of PM1183, a novel drug candidate which induces double-stranded DNA breaks to cause cell death.

"PM1183 is an exciting compound, and we are extremely pleased to be working with Pharma Mar on this project," said Peter Meldrum, President and Chief Executive Officer of Myriad Genetics, Inc. "We believe this agreement, as well as our many other companion diagnostic projects for DNA-damaging agents, demonstrates the importance of a patient's BRCA status in identifying potential responders to this class of drugs."

Under the agreement, Myriad will assess the BRCA status in patients who respond to PM1183. Myriad has entered into similar agreements with Abbott Pharmaceuticals, Astra Zeneca, BioMarin Pharmaceuticals and Cephalon to provide companion diagnostic testing with the BRACAnalysis(R) test for clinical trial enrollment.

About BRACAnalysis(R)

BRACAnalysis is a molecular diagnostic test developed by Myriad Genetics which analyzes the BRCA1 and BRCA2 genes to assess a woman's risk for developing breast and ovarian cancer and guide treatment decisions for women with hereditary breast and ovarian cancer. A woman who tests positive with the BRACAnalysis test has, on average, up to an 86% risk of developing breast cancer during her lifetime and up to a 44% risk of developing ovarian cancer. BRACAnalysis provides important information that the Company believes will help patients and their physicians make better informed lifestyle, surveillance, and preventive medication and treatment decisions. For more information about BRACAnalysis, please call 1-800-4-MYRIAD, or visit http://www.myriad.com.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patient's lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's portfolio of molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com

Myriad, the Myriad logo, BRACAnalysis, Colaris, Colaris AP, Melaris, TheraGuide, Prezeon, OnDose, Panexia and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries. MYGN-G

Safe Harbor Statement

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to testing to be conducted by Myriad under the agreement with Pharma Mar and the Company's strategic directives under the caption "About Myriad Genetics". These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our existing molecular diagnostic tests and companion diagnostic services may decline or will not continue to increase at historical rates; the risk that we may be unable to expand into new markets outside of the United States; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and companion diagnostic services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and companion diagnostic services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and companion diagnostic services and any future products are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with manufacturing our products or operating our laboratory testing facilities; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of healthcare payment systems; risks related to our ability to obtain new corporate collaborations and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we acquire; the development of competing tests and services; the risk that we or our licensors may be unable to protect the proprietary technologies underlying our tests; the risk of patent-infringement and invalidity claims or challenges of our patents; risks of new, changing and competitive technologies and regulations in the United States and internationally; and other factors discussed under the heading "Risk Factors" contained in Item 1A in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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Myriad Genetics and Pharma Mar Enter Into Companion Diagnostic Agreement

SAN DIEGO, July 26, 2012 /PRNewswire/ -- Cardium Therapeutics (NYSE MKT: CXM) today announced the publication of preclinical findings demonstrating that cardiac ischemia plays an important role in adenovector gene delivery (transfection) in mammalian hearts. The new findings were published in the peer-reviewed journal Human Gene Therapy Methods in an article entitled "Ischemia-Reperfusion Increases Transfection Efficiency of Intracoronary Adenovirus type 5 in Pig Heart in Situ," which is available online at http://online.liebertpub.com/doi/full/10.1089/hgtb.2012.048.

(Logo: http://photos.prnewswire.com/prnh/20051018/CARDIUMLOGO)

The published findings demonstrate that Cardium's innovative technique employing transient cardiac ischemia can be used to dramatically enhance gene delivery and transfection efficiency after one-time intracoronary administration of adenovector in mammalian hearts. Two consecutive but brief periods of coronary artery occlusion combined with co-administration of nitroglycerin increased both adenovector presence (measured by PCR) and transgene expression (assessed by luciferase activity) by over two orders of magnitude (>100 fold) in the heart, as compared to prior intracoronary artery delivery methods.

"The clinical success of DNA-based therapies can be enhanced by employing optimized gene delivery methods," stated Dr. Gabor M. Rubanyi, Cardium's Chief Scientific Officer and co-author of the published paper. "In addition, data analysis from the AGENT 1 through 4 clinical studies, involving more than 650 patients in Phase 1/2 through Phase 2/3, showed that patients with more severe forms of coronary artery disease which is associated with increased ischemia tended to be more responsive to the one-time administration of Generx than patients with less severe disease. The research results published in Human Gene Therapy Methods extend those findings and demonstrate that Cardium's new technique for adenovector gene delivery in the heart can be used to dramatically boost adenovector delivery. By enhancing uptake even in patients with less severe forms of disease and ischemia, it would be expected to reduce response variability and allow for the potential treatment of patients with a broader range of associated coronary artery disease. The new treatment protocols for Cardium's recently-initiated ASPIRE clinical study have been developed to use our knowledge about induced transient ischemia techniques to leverage these research findings and enhance the non-surgical, catheter-based delivery of Generx to the heart," stated Dr. Rubanyi.

Cardium's new method of adenovector delivery to the heart takes advantage of the fact that transient ischemia may reduce the permeability barrier of the vascular endothelium and may increase the number of available coxsackie-adenovirus receptors mediating adenovector uptake. Balloon angioplasty catheters have been used for many years to dilate blocked coronary arteries, sometimes with use of a stent, and these catheters have also been used safely by cardiologists in patients with coronary artery disease to study the effects of brief ischemia. Cardium's new technique inflates the balloon in non-narrowed coronary artery areas, just enough to briefly interrupt flow using inflation pressure that is significantly less than that used for performing routine angioplasty procedures.

Cardium's recently initiated Russian-based ASPIRE Phase 3 registration study of patients with chronic myocardial ischemia and advanced angina pectoris uses transient ischemia techniques during non-surgical percutaneous catheterization with a standard angioplasty catheter together with the intracoronary infusion of nitroglycerin with the Generx [Ad5FGF-4] product candidate. The Company's Generx product candidate is intended to stimulate the growth of collateral blood vessels to effectively bypass coronary artery atherosclerotic blockages without surgical procedures or angioplasty and stents.

The studies published in Human Gene Therapy Methods were conducted at Emory University School of Medicine by Jakob Vinten-Johansen, Ph.D. and colleagues, and were co-sponsored by a Small Business Innovation Research grant from the National Institutes of Health (Cardium Therapeutics) and the Carlyle Fraser Heart Center (Emory). A presentation titled: "New Perspectives for Angiogenic Gene Therapy to Treat Myocardial Ischemia in Patients with Coronary Disease" was presented at the 2012 American Society of Gene & Cell Therapy Meeting in May 2012 and is available for viewing at http://www.cardiumthx.com/pdf/Generx-ASGCT-May-2012-Rubanyi.pdf. At the conference, Cardium also presented a late-breaking poster titled "Transient Ischemia is Necessary for Efficient Adenovector Gene Transfer in the Heart". The poster presentation can be viewed at http://www.cardiumthx.com/pdf/Generx-ASGCT-Poster-Presentation-May-2012.pdf.

About Generx and the ASPIRE Study

Generx (Ad5FGF-4) is a disease-modifying regenerative medicine biologic that is being developed to offer a one-time, non-surgical option for the treatment of myocardial ischemia in patients with stable angina due to coronary artery disease, who might otherwise require surgical and mechanical interventions, such as coronary artery by-pass surgery or balloon angioplasty and stents. Similar to surgical/mechanical revascularization approaches, the goal of Cardium's Generx product candidate is to improve blood flow to the heart muscle but to do so non-surgically, following a single administration from a standard balloon angioplasty catheter. The video "Cardium Generx Cardio-Chant" provides an overview Generx and can be viewed at http://www.youtube.com/watch?v=pjUndFhJkjM.

In March 2012, Cardium reported on the ASPIRE Phase 3 registration study to evaluate the therapeutic effects of its lead product candidate Generx in patients with myocardial ischemia due to coronary artery disease. The ASPIRE study, a 100-patient, randomized and controlled multi-center study to be conducted at up to eight leading cardiology centers in the Russian Federation, is designed to further evaluate the safety and effectiveness of Cardium's Generx DNA-based angiogenic product candidate, which has already been tested in clinical studies involving 650 patients at more than one hundred medical centers in the U.S., Europe and elsewhere. The efficacy of Generx will be quantitatively assessed using rest and stress SPECT (Single-Photon Emission Computed Tomography) myocardial imaging to sensitively measure improvements in microvascular cardiac perfusion following a one-time, non-surgical, catheter-based administration of Generx. A recent article, "Cardium's Heart Disease Gene Therapy Advancing with New Discoveries," outlining the history of the Generx clinical development program is available at http://sandiegobiotechnology.com/topics/4705/cardiums-heart-disease-gene-therapy-moving-toward-commercialization/.

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Published Findings In Human Gene Therapy Methods Journal Demonstrate Cardium's New Catheter-Based Method Significantly ...

WASHINGTON Researchers have for the first time used gene therapy to restore normal hearing to mice that were born deaf. The findings could lead to a successful therapy for people with congenital hearing loss.

Congenital hearing loss or deafness from birth is one of the most common sensory deficits. It is often the result of genetic defects that affect the proper functioning of hair cells inside the inner ear. The hair cells, which move in response to sound vibration, transmit auditory signals to the brain. Experts believe there may be as many as 100 genes whose mutation can cause some form of hearing loss. Researchers created a mouse model of congenital deafness in humans by breeding rodents that lacked the gene that makes vesicular glutamate transporter-3, or VGLUT3 for short - an essential protein that enables hair cells in the ear to receive and send auditory signals. People with a defective copy of the VGLUT-3 gene suffer diminished hearing over time. Because the experimental mice didnt have the gene at all, their deafness was profound.

Lawrence Lustig, an otolaryngologist or hearing specialist at the University of California San Francisco, says researchers used a virus that doesnt cause illness in humans to carry a corrected copy of the VGLUT3 gene into the ear canals of the deaf mice.

Then, they sealed up the tiny hole they had made in a membrane in the rodents inner ears to inject the virus, and waited. The first time I saw the results, I didnt believe it. The hearing looked essentially normal in these mice, Lustig said. Lustig says that within a week, the mice showed the first signs that they could hear. By the second week, the rodents' hearing was essentially normal, as measured by a stimulation test in which researchers played tones and then monitored the rodents brain response to the sounds.

The researchers treated both newborn and adult mice with the therapy. Hearing lasted one-and-a-half years in the grown rodents and at least nine months in the newborns. Mice typically live two years. Current methods of treating congenital deafness include hearing amplification or hearing aids and cochlear implants, which are surgically embedded in the skull. But the devices often produce audio distortion or sound levels that are too low.

If deafness is caused by a defective gene, Lustig says, gene therapy as demonstrated by the experiment with VGLUT3 has the potential to cure many forms of hearing loss. Our next steps that we are actually working on right now are to take a much more common form of genetic hearing loss [caused by a different gene] and then trying that again in mice and see if it works. And if that can work, then the next steps are see how we can implement this in kids who are born with deafnes, Lustig said.

An article by the University of Californias Lawrence Lustig and colleagues on the use of gene therapy to cure congenital hearing loss is published in the journal Cell Press.

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Researchers Cure Mouse Deafness with Gene Therapy

Peter Aldhous, San Francisco bureau chief

It's official: stem cells are drugs. At least, that's the opinion of the US District Court in Washington DC, which has ruled that the Food and Drug Administration (FDA) has the authority to regulate clinics offering controversial stem cell therapies.

Treatments in which stem cells are harvested from bone marrow and injected straight back into the same patient are deemed part of routine medical practice - not regulated by the US government. But if the cells are subjected to more than "minimal manipulation", the FDA maintains that the therapy becomes a "drug", which must be specifically approved for use.

Christopher Centeno, medical director of Regenerative Sciences, vows to appeal. "This is really round one," he says. "Our position remains that a patient's cells are not drugs."

Scott hopes that the FDA will now step up its efforts to regulate other clinics offering unproven stem cell therapies. These include Celltex of Sugar Land, Texas, which rose to prominence after Texas governor Rick Perry was injected with stem cells supplied by the company to aid his recovery from back surgery.

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Ruling frees FDA to crack down on stem cell clinics

ScienceDaily (July 25, 2012) In the days following the 2010 Deepwater Horizon oil spill, methane-eating bacteria bloomed in the Gulf of Mexico, feasting on the methane that gushed, along with oil, from the damaged well. The sudden influx of microbes was a scientific curiosity: Prior to the oil spill, scientists had observed relatively few signs of methane-eating microbes in the area.

Now researchers at MIT have discovered a bacterial gene that may explain this sudden influx of methane-eating bacteria. This gene enables bacteria to survive in extreme, oxygen-depleted environments, lying dormant until food -- such as methane from an oil spill, and the oxygen needed to metabolize it -- become available. The gene codes for a protein, named HpnR, that is responsible for producing bacterial lipids known as 3-methylhopanoids. The researchers say producing these lipids may better prepare nutrient-starved microbes to make a sudden appearance in nature when conditions are favorable, such as after the Deepwater Horizon accident.

The lipid produced by the HpnR protein may also be used as a biomarker, or a signature in rock layers, to identify dramatic changes in oxygen levels over the course of geologic history.

"The thing that interests us is that this could be a window into the geologic past," says MIT postdoc Paula Welander, who led the research. "In the geologic record, many millions of years ago, we see a number of mass extinction events where there is also evidence of oxygen depletion in the ocean. It's at these key events, and immediately afterward, where we also see increases in all these biomarkers as well as indicators of climate disturbance. It seems to be part of a syndrome of warming, ocean deoxygenation and biotic extinction. The ultimate causes are unknown."

Welander and Roger Summons, a professor of Earth, atmospheric and planetary sciences, have published their results this week in the Proceedings of the National Academy of Sciences.

A sign in the rocks

Earth's rocky layers hold remnants of life's evolution, from the very ancient traces of single-celled organisms to the recent fossils of vertebrates. One of the key biomarkers geologists have used to identify the earliest forms of life is a class of lipids called hopanoids, whose sturdy molecular structure has preserved them in sediment for billions of years. Hopanoids have also been identified in modern bacteria, and geologists studying the lipids in ancient rocks have used them as signs of the presence of similar bacteria billions of years ago.

But Welander says hopanoids may be used to identify more than early life forms: The molecular fossils may be biomarkers for environmental phenomena -- such as, for instance, periods of very low oxygen.

To test her theory, Welander examined a modern strain of bacteria called Methylococcus capsulatus, a widely studied organism first isolated from an ancient Roman bathhouse in Bath, England. The organism, which also lives in oxygen-poor environments such as deep-sea vents and mud volcanoes, has been of interest to scientists for its ability to efficiently consume large quantities of methane -- which could make it helpful in bioremediation and biofuel development.

For Welander and Summons, M. capsulatus is especially interesting for its structure: The organism contains a type of hopanoid with a five-ring molecular structure that contains a C-3 methylation. Geologists have found that such methylations in the ring structure are particularly well-preserved in ancient rocks, even when the rest of the organism has since disappeared.

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Newfound gene may help bacteria survive in extreme environments: Resulting microbial lipids may also signify oxygen ...

Public release date: 25-Jul-2012 [ | E-mail | Share ]

Contact: Mary F. Masson mfmasson@umich.edu 734-764-2220 University of Michigan Health System

Ann Arbor, Mich. University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.

About 50% of congenital myopathy cases currently do not have a known genetic basis, presenting a clear barrier to understanding disease and developing therapy, says James Dowling, M.D., Ph.D., the paper's co-senior author and assistant professor of Pediatric Neurology at the University of Michigan's C.S. Mott Children's Hospital. Finding a new myopathy gene opens the possibility of providing a genetic explanation for disease in these individuals where no genetic cause is currently known.

In addition, "the identification of a new myopathy gene is an essential first step towards understanding why this disease occurs and how we combat its effects." says Dowling, who worked with Margit Burmeister, Ph.D. and her team from the University of Michigan's Molecular and Behavioral Neuroscience Institute to study the new myopathy gene (CCDC78).

Dowling says the gene, which has not been studied previously, is an important potential regulator of muscle function and, in particular, part of an important muscle structure called the triad.

"Many myopathies and dystrophies have abnormal triad structure/function, so finding a new gene product involved in its regulation will help researchers better understand the triad and its relationship to muscle disease," Dowling says.

Congenital myopathies are clinically and genetically heterogeneous diseases that typically become evident in childhood with hypotonia and weakness. They are associated with impaired mobility, progressive scoliosis, chronic respiratory failure and often early death.

Currently there are no known treatments or disease modifying therapies for congenital myopathies.

The researchers performed linkage analysis followed by whole exome capture and next generation sequencing in a family with congenital myopathy. They then validated the gene mutation and provided insights into the disease pathomechanisms using the zebrafish model system.

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Researchers find new gene mutation associated with congenital myopathy

ScienceDaily (July 25, 2012) University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.

About 50% of congenital myopathy cases currently do not have a known genetic basis, presenting a clear barrier to understanding disease and developing therapy, says James Dowling, M.D., Ph.D., the paper's co-senior author and assistant professor of Pediatric Neurology at the University of Michigan's C.S. Mott Children's Hospital. Finding a new myopathy gene opens the possibility of providing a genetic explanation for disease in these individuals where no genetic cause is currently known.

In addition, "the identification of a new myopathy gene is an essential first step towards understanding why this disease occurs and how we combat its effects." says Dowling, who worked with Margit Burmeister, Ph.D. and her team from the University of Michigan's Molecular and Behavioral Neuroscience Institute to study the new myopathy gene (CCDC78).

Dowling says the gene, which has not been studied previously, is an important potential regulator of muscle function and, in particular, part of an important muscle structure called the triad.

"Many myopathies and dystrophies have abnormal triad structure/function, so finding a new gene product involved in its regulation will help researchers better understand the triad and its relationship to muscle disease," Dowling says.

Congenital myopathies are clinically and genetically heterogeneous diseases that typically become evident in childhood with hypotonia and weakness. They are associated with impaired mobility, progressive scoliosis, chronic respiratory failure and often early death.

Currently there are no known treatments or disease modifying therapies for congenital myopathies.

The researchers performed linkage analysis followed by whole exome capture and next generation sequencing in a family with congenital myopathy. They then validated the gene mutation and provided insights into the disease pathomechanisms using the zebrafish model system.

Dowling says the researchers' next step is to further model the disease using zebrafish, in the hopes that this knowledge can be translated into therapy development.

"The study provides the first descriptions of the zebrafish model, and gives insight into how we will use it," says Dowling, who also is director of the Pediatric Neuromuscular Disorders Clinic at C.S. Mott Children's Hospital.

See more here:
New gene mutation associated with congenital myopathy

By Lynne Friedmann

A genetic test to predict the risk for prostate cancer could reduce the need for repeat biopsies in men who have previously had negative biopsies.

In a clinical trial, 1,654 men who had prostate biopsies also had genetic studies conducted that looked for the presence of genetic variations that may have an association with prostate cancer risk.

The genetic test outperformed the widely used PSA (Prostate-Specific Antigen) test in assessing cancer risk. Because this genetic score is available at any time in a mans lifetime it could be used as a pre-screening test thus leaving aggressive PSA screening only to men at higher genetic risk.

The goal is to avoid, particularly in older men, unnecessary repeat biopsy procedures which carry with them the risk of infection and potential hospitalizations.

Findings appear in the journal of European Urology. News release at http://bit.ly/M7iaHV

Inhibiting malaria parasite development

Malaria is responsible worldwide for more than 1.2 million human deaths annually. Severe forms of the disease are caused by the parasite Plasmodium falciparum transmitted to humans by the bite of female Anopheles mosquitoes. Lack of vaccines, together with the parasites ability to develop drug resistance, has thwarted eradication efforts.

An international team of scientists, led by researchers from the Department of Pediatrics at the UC School of Medicine, has identified the first reported inhibitors of a key enzyme essential for the development and survival of P. falciparum even in parasites that developed resistance to currently available drugs.

People with a natural deficiency in this enzyme are protected from malaria and its deadly symptoms, an observation that triggered the research effort. The hope is the discovery could provide the basis for future anti-malarial drug design.

Original post:
New genetic test predicts prostate cancer risk

ScienceDaily (July 25, 2012) A University of Sheffield academic is helping a team of citizen scientists to carry out crucial research into European genetic heritage.

Citizen Scientists are not required to have a scientific background or training, but instead they possess a passion for the subject and are increasingly being empowered by the scientific community to get involved in research.

Dr Andy Grierson, from the University of Sheffield's Institute for Translational Neuroscience (SITraN), has helped a team of citizen scientists from Europe and North America to identify vital new clues to tell the story of Europe's genetic history.

Dr Grierson explained: "Understanding European history since man first arrived on the continent is a huge challenge for archaeologists and historians.

"One way that scientists can help is by studying the genetics of European men. All men carry a Y chromosome that they inherit from their father, which has been passed down the generations from father to son for thousands of years. So most men in Europe will share common ancestry at some point in the past, and we are able to investigate this shared ancestry using genetic studies of the Y chromosome.

"However, up until recently, there have not been many genetic clues on the Y chromosome to allow scientists to be certain about identifying different populations."

The team has addressed this problem by downloading human genome data obtained by the 1000 Genomes Project from the Sanger Centre in Cambridge. Then, working on their home computers, they managed to extract 200 novel genetic variants from Y chromosomes of the most numerous group of western European men.

By determining the patterns of these markers in each of the 1000 Genomes Project samples, the team was able to draw up a new family tree for the majority of men in Western Europe.

The group hopes that this resource will allow a much more detailed analysis of migration and expansion of populations in Europe. For example, some of the new genetic markers may help to study the origins and movements of different historical and cultural groups such as the Celts.

Dr Grierson added: "This community-led approach to genetic research could easily be adopted by other research areas. In particular, the 1000 Genomes Project has made the whole genome sequence of more than 2,000 individuals freely available for research purposes. These sequences potentially contain new information that will give important insight in diverse disciplines such as clinical medicine and evolutionary biology.

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Citizen researchers help unlock European genetic heritage

ScienceDaily (July 25, 2012) A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published in Human Gene Therapy.

The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zo Coulombe, and Joel Rousseau, Laval University, Quebec,and University of Quebec, Canada, takes advantage of the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. As a model of how this method could be used to treat genetic disease, the authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich's ataxia. The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, conclude the authors.

"This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia

Public release date: 25-Jul-2012 [ | E-mail | Share ]

Contact: Mary Beth O'Leary moleary@cell.com 617-397-2802 Cell Press

A new gene therapy approach can reverse hearing loss caused by a genetic defect in a mouse model of congenital deafness, according to a preclinical study published by Cell Press in the July 26 issue of the journal Neuron. The findings present a promising therapeutic avenue for potentially treating individuals who are born deaf.

"This is the first time that an inherited, genetic hearing loss has been successfully treated in laboratory mice, and as such represents an important milestone for treating genetic deafness in humans," says senior study author Lawrence Lustig of the University of California, San Francisco.

Hearing loss is one of the most common human sensory deficits, and it results from damage to hair cells in the inner ear. About half of the cases of congenital hearing loss are caused by genetic defects. However, the current treatment optionshearing amplification devices and cochlear implantsdo not restore hearing to normal levels. Correcting the underlying genetic defects has the potential to fully restore hearing, but previous attempts to reverse hearing loss caused by genetic mutations have not been successful.

Addressing this challenge in the new study, Lustig and his team used mice with hereditary deafness caused by a mutation in a gene coding for a protein called vesicular glutamate transporter-3 (VGLUT3). This protein is crucial for inner hair cells to send signals that enable hearing. Two weeks after the researchers delivered the VGLUT3 gene into the inner ear through an injection, hearing was restored in all of the mice. This improvement lasted between seven weeks and one and a half years when adult mice were treated, and at least nine months when newborn mice received the treatment.

The therapy did not damage the inner ear, and it even corrected some structural defects in the inner hair cells. Because the specific gene delivery method used is safe and effective in animals, the findings hold promise for future human studies. "For years, scientists have been hinting at the possibility of gene therapy as a potential cure for deafness," Lustig says. "In this study, we now provide a very real and big step towards that goal."

###

Akil et al.: "Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally-Mediated Gene Therapy."

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Gene therapy holds promise for reversing congenital hearing loss

Public release date: 24-Jul-2012 [ | E-mail | Share ]

Contact: Mount Sinai Press Office newsmedia@mssm.edu 212-241-9200 The Mount Sinai Hospital / Mount Sinai School of Medicine

Researchers from Mount Sinai School of Medicine have developed a new computational method that will make it easier for scientists to identify and prioritize genes, drug targets, and strategies for repositioning drugs that are already on the market. By mining large datasets more simply and efficiently, researchers will be able to better understand gene-gene, protein-protein, and drug/side-effect interactions. The new algorithm will also help scientists identify fellow researchers with whom they can collaborate.

Led by Avi Ma'ayan, PhD, Assistant Professor of Pharmacology and Systems Therapeutics at Mount Sinai School of Medicine, and Neil Clark, PhD a postdoctoral fellow in the Ma'ayan laboratory, the team of investigators used the new algorithm to create 15 different types of gene-gene networks. They also discovered novel connections between drugs and side effects, and built a collaboration network that connected Mount Sinai investigators based on their past publications.

"The algorithm makes it simple to build networks from data," said Dr. Ma'ayan. "Once high dimensional and complex data is converted to networks, we can understand the data better and discover new and significant relationships, and focus on the important features of the data."

The group analyzed one million medical records of patients to build a network that connects commonly co-prescribed drugs, commonly co-occurring side effects, and the relationships between side effects and combinations of drugs. They found that reported side effects may not be caused by the drugs, but by a separate condition of the patient that may be unrelated to the drugs. They also looked at 53 cancer drugs and connected them to 32 severe side effects. When chemotherapy was combined with cancer drugs that work through cell signaling, there was a strong link to cardiovascular related adverse events. These findings can assist in post-marketing surveillance safety of approved drugs.

The approach is presented in two separate publications in the journals BMC Bioinformatics and BMC Systems Biology. The tools that implement the approach Genes2FANs and Sets2Networks can be found online at http://actin.pharm.mssm.edu/genes2FANs and http://www.maayanlab.net/S2N.

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Research in the Ma'ayan laboratory is funded by the National Institutes of Health.

About The Mount Sinai Medical Center

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New method to find novel connections from gene to gene, drug to drug and between scientists

25.07.2012 - (idw) Medizinische Hochschule Hannover

Ascenion assists the project from patenting to commercialization 25 July 2012, Munich, Hannover and Laupheim The Hannover Medical School (MHH) and Rentschler Biotechnologie GmbH have signed a licensing agreement for the commercialization of a vector system for gene therapy that promises significant therapeutic and economic advantages over conventional technologies. Rentschler, an experienced specialist in the development of cell culture-based manufacturing processes and the production of relevant biopharmaceuticals, attains the right to develop packaging cell lines containing the novel vector and to use these commercially for the contract manufacturing of gene therapeutics.

Scientists at the MHH previously showed that the vector system can be flexibly adapted to various applications and is produced efficiently in cell lines normally used in the production of biopharmaceuticals. Ascenion, the MHHs exclusive technology transfer partner, facilitated the commercialization of the invention, from the patent application to forging the cooperation and negotiating the licensing agreement.

The MHH vector represents an advancement over conventional vector systems, whose therapeutic efficacy has already been established in many indications. We have created a new platform with broad applications in gene therapy that offers improved safety to patients and increased cost-effectiveness to developers and healthcare systems, says Prof. Christopher Baum, Director of the Institute for Experimental Haematology at the MHH. Prof. Baum developed the vector system together with his colleagues Dr Julia Srth and Dr Dr Axel Schambach, supported in part by funding from the Deutsche Forschungsgemeinschaft (DFG) Cluster of Excellence REBIRTH (from Regenerative Biology to Reconstructive Therapy).

The main areas of application range from introducing therapeutic genes into blood stem cells and lymphocytes in order to correct congenital defects, to improving cellular immunity in cancer and HIV infection. Further cell types and diseases are also being targeted using the new vector system.

Prof. Roland Wagner, Senior Vice President Development at Rentschler Biotechnologie GmbH, emphasizes: The new vector system complements our spectrum for the development of virus-based biologics used in gene therapy and virotherapy, which, beside Rentschlers own AAV vector technology, will be made available to our clients for the development of these products. Rentschler is currently financing further research at the MHH on highly efficient and stable packaging cell lines for retroviral vectors.

Ascenion will continue to assist with the project. We are delighted that the research findings from the MHH have been developed to the benefit of patients, commented Dr Christian Stein, Ascenions CEO. The partners complement each other perfectly: the MHH contributes its innovative technology and proximity to patients, and Rentschler contributes its longstanding experience in the production of biopharmaceuticals and its tight industry network.

Contacts Ascenion: Anja Kroke, Corporate Communications T: +49 (0)89 318814-30, E: kroke@ascenion.de Ascenion GmbH, Herzogstrae 64, 80803 Munich

MHH: Camilla Krause, Public Relations Cluster of Excellence REBIRTH T: +49 (0)511 532-6793, E: pressestelle@mh-hannover.de

Rentschler Biotechnologie: Friederike Braun, Director Marketing T: +49 (0)7392 701-478 , E: friederike.braun@rentschler.de Rentschler Biotechnologie GmbH, Erwin-Rentschler-Str. 21, 88471 Laupheim

Continued here:
MHH and Rentschler Sign Licensing Agreement for an Innovative Gene Therapy Vector System

ScienceDaily (July 24, 2012) Researchers at the Royal College of Surgeons in Ireland (RCSI) have developed a new method of repairing bone using synthetic bone graft substitute material, which combined with gene therapy, can mimic real bone tissue and has potential to regenerate bone in patients who have lost large areas of bone from either disease or trauma.

The researchers have developed an innovative scaffold material (made from collagen and nano-sized particles of hydroxyapatite) which acts as a platform to attract the body's own cells and repair bone in the damaged area using gene therapy. The cells are tricked into overproducing bone producing proteins known as BMPs, encouraging regrowth of healthy bone tissue. This is the first time these in-house synthesised nanoparticles have been used in this way and the method has potential to be applied to regenerate tissues in other parts of the body.

Professor Fergal O'Brien, Principal Investigator on the project explained: "Previously, synthetic bone grafts had proven successful in promoting new bone growth by infusing the scaffold material with bone producing proteins. These proteins are already clinically approved for bone repair in humans but concerns exist that the high doses of protein required in clinical treatments may potentially have negative side effects for the patient such as increasing the risk of cancer. Other existing gene therapies use viral methods which also carry risks."

"By stimulating the body to produce the bone-producing protein itself, using non-viral methods these negative side effects can be avoided and bone tissue growth is promoted efficiently and safely," Professor O'Brien said.

The research is the result of a collaborative project carried out between the Tissue Engineering Research Group led by Professor Fergal O'Brien and Dr. Garry Duffy in the Department of Anatomy, RCSI; Professor Kazuhisa Bessho, Kyoto University, Japan, and Dr. Glenn Dickson, Queen's University Belfast, Northern Ireland and consists of a multi-disciplinary research effort between cell biologists, clinicians and engineers. Results of this study were recently published in the journal Advanced Materials with Dr. Caroline Curtin, a postdoctoral researcher in the Department of Anatomy, RCSI, as first author.

Bone grafts are second only to blood transfusions on the list of transplanted materials worldwide with approximately 2.2 million procedures performed annually (1) at an estimated cost of $2.5 billion per year (2). At present, the majority of these procedures involve either transplanting bone from another part of the patient's own body (autograft) or from a donor (allograft). However, these procedures have a number of risks such as infections or the bone not growing properly at the area of transplantation. Therefore there is a large potential market for bone graft substitute materials such as the innovative scaffolds being developed by the RCSI team and their collaborators.

While the biomaterials developed in this project have undoubted potential for bone repair with the capability to act as a superior alternative to existing bone graft treatments, this gene delivery platform may also have significant potential in the regeneration of other degenerated or diseased tissues in the body when combined with different therapeutic genes. This is currently a major focus of ongoing research in the Tissue Engineering Research Group which has a particular interest in using the platform to deliver genes that promote the formation of blood vessels (using angiogenic genes) in the regeneration of tissues which suffer from compromised blood supply such as heart wall tissue which has been damaged following a heart attack.

This research was funded by the European Research Council under the European Community's Seventh Framework Programme and a Science Foundation Ireland, President of Ireland Young Researcher Award.

(1) Lewandrowski KU, Gresser JD, Wise DL, Trantol DJ. Bioresorbable bone graft substitutes of different osteoconductivities: a histologic evaluation of osteointegration of poly(propylene glycol-co-fumaric acid)-based cement implants in rats. Biomaterials 2000; 21:757-764.

(2) Desai BM (2007) Osteobiologics. Am J Orthop (Belle Mead NJ) 2007; 36:8-11.

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Regenerating bone tissue using gene therapy: New method may work with other tissues as well

25 July 2012 Last updated at 00:05 ET By Susan Watts Newsnight Science editor, BBC News

Scientists behind Olympic Team GB are working on genetic tests to understand why some athletes are prone to injury, BBC's Newsnight has learned.

Tendon injuries and stress fractures are common in elite athletes, but how and why they happen is less clear.

University College London's Prof Hugh Montgomery says they have found a gene they think strongly influences the risk of stress fracture and more will come.

It is hoped the research will allow training to be individually tailored.

Diet, repetitive strain and loading are all known to play a part, and scientists say there is clearly a strong genetic element.

Director of the Institute for Human Health and Performance at University College London, Prof Montgomery carried out groundbreaking work on genes and fitness in the 1990s, most notably the "ACE" gene, thought to be linked to endurance.

"If we understood that genetic component we would have a much better understanding of the patho-physiology - the disease processes that let that happen," says Prof Montgomery.

He has been working closely with the English Institute of Sport (EIS), which aims to apply the latest in sports science and medicine for the benefit of Britain's Olympic and Paralympic athletes.

EIS's Director of Sport Science, Dr Ken van Someren, told Newsnight he is keen to apply the latest genetics discoveries.

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Team GB trials injury gene tests

Public release date: 25-Jul-2012 [ | E-mail | Share ]

Contact: Caroline McCall cmccall5@mit.edu Massachusetts Institute of Technology

CAMBRIDGE, Mass. -- In the days following the 2010 Deepwater Horizon oil spill, methane-eating bacteria bloomed in the Gulf of Mexico, feasting on the methane that gushed, along with oil, from the damaged well. The sudden influx of microbes was a scientific curiosity: Prior to the oil spill, scientists had observed relatively few signs of methane-eating microbes in the area.

Now researchers at MIT have discovered a bacterial gene that may explain this sudden influx of methane-eating bacteria. This gene enables bacteria to survive in extreme, oxygen-depleted environments, lying dormant until food such as methane from an oil spill, and the oxygen needed to metabolize it become available. The gene codes for a protein, named HpnR, that is responsible for producing bacterial lipids known as 3-methylhopanoids. The researchers say producing these lipids may better prepare nutrient-starved microbes to make a sudden appearance in nature when conditions are favorable, such as after the Deepwater Horizon accident.

The lipid produced by the HpnR protein may also be used as a biomarker, or a signature in rock layers, to identify dramatic changes in oxygen levels over the course of geologic history.

"The thing that interests us is that this could be a window into the geologic past," says MIT postdoc Paula Welander, who led the research. "In the geologic record, many millions of years ago, we see a number of mass extinction events where there is also evidence of oxygen depletion in the ocean. It's at these key events, and immediately afterward, where we also see increases in all these biomarkers as well as indicators of climate disturbance. It seems to be part of a syndrome of warming, ocean deoxygenation and biotic extinction. The ultimate causes are unknown."

Welander and Roger Summons, a professor of Earth, atmospheric and planetary sciences, have published their results this week in the Proceedings of the National Academy of Sciences.

A sign in the rocks

Earth's rocky layers hold remnants of life's evolution, from the very ancient traces of single-celled organisms to the recent fossils of vertebrates. One of the key biomarkers geologists have used to identify the earliest forms of life is a class of lipids called hopanoids, whose sturdy molecular structure has preserved them in sediment for billions of years. Hopanoids have also been identified in modern bacteria, and geologists studying the lipids in ancient rocks have used them as signs of the presence of similar bacteria billions of years ago.

But Welander says hopanoids may be used to identify more than early life forms: The molecular fossils may be biomarkers for environmental phenomena such as, for instance, periods of very low oxygen.

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Newfound gene may help bacteria survive in extreme environments

Public release date: 25-Jul-2012 [ | E-mail | Share ]

Contact: Amy Stone a.f.stone@sheffield.ac.uk 01-142-221-046 University of Sheffield

A University of Sheffield academic is helping a team of citizen scientists to carry out crucial research into European genetic heritage.

Citizen Scientists are not required to have a scientific background or training, but instead they possess a passion for the subject and are increasingly being empowered by the scientific community to get involved in research.

Dr Andy Grierson, from the University of Sheffield's Institute for Translational Neuroscience (SITraN), has helped a team of citizen scientists from Europe and North America to identify vital new clues to tell the story of Europe's genetic history.

Dr Grierson explained: "Understanding European history since man first arrived on the continent is a huge challenge for archaeologists and historians.

"One way that scientists can help is by studying the genetics of European men. All men carry a Y chromosome that they inherit from their father, which has been passed down the generations from father to son for thousands of years. So most men in Europe will share common ancestry at some point in the past, and we are able to investigate this shared ancestry using genetic studies of the Y chromosome.

"However, up until recently, there have not been many genetic clues on the Y chromosome to allow scientists to be certain about identifying different populations."

The team has addressed this problem by downloading human genome data obtained by the 1000 Genomes Project from the Sanger Centre in Cambridge. Then, working on their home computers, they managed to extract 200 novel genetic variants from Y chromosomes of the most numerous group of western European men.

By determining the patterns of these markers in each of the 1000 Genomes Project samples, the team was able to draw up a new family tree for the majority of men in Western Europe.

Continued here:
Citizen science helps unlock European genetic heritage

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/j69n56/genetic_testing_ma) has announced the addition of the "Genetic Testing Market Report: 2012 Edition" report to their offering.

The genetic testing market segment represents the highest growth opportunity compared to other segments within the molecular diagnostics market. The growth of the market is backed by increasing prevalence of genetic diseases, aging population, and rising awareness. Vast application areas including newborn screening, prenatal and preimplantation testing is also expected to add to the future growth of the market.

The technological advancements of genetic testing are expected to break new grounds in the field of personalized medicine as genetic testing helps not only in the better diagnosis of genetic diseases but also aids in selecting the best medication depending on the DNA structure of the patient to ensure superior drug response.

Direct to customer (DTC) genetic testing is one of the major trends in the market, which is expected to bring positive changes in the popularity of genetic testing. However, the quality, accuracy, and proper interpretation of test results of DTC genetic testing continue to challenge the future growth of the market. Furthermore, the risk of genetic discrimination and psychological consequences of genetic testing are also expected to hinder the growth of the market in the coming years.

Publication Overview:

The current report analyzes the genetic testing market. It discusses the drivers and challenges prevailing in the genetic testing market. It presents the competitive conditions prevailing in the market, along with the profiles of major players.

Key Topics Covered:

Genetic Testing: An Introduction

- Types of Genetic Testing

Excerpt from:
Research and Markets: Genetic Testing Market Report: 2012 Edition