Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

AMT inks agreement with Progenika Biopharma for development of LPLchip

Amsterdam Molecular Therapeutics (AMT) and Progenika Biopharma announced today that the companies have entered into a development and commercialization agreement for LPLchip(TM), a diagnostic tool to rapidly diagnose patients with complete and partial lipoprotein lipase deficiency (LPLD). AMT has developed Glybera(R) as a gene therapy for patients with this disease.

29 Oct 2009

New anti-cancer medications may help in managing cancerous conditions

Bold new strategies in the battle against cancer may turn forms of the disease that presently are incurable into manageable conditions that can be controlled for long periods of time, according to an article in the current issue of Chemical & Engineering News, ACS' weekly newsmagazine.

29 Oct 2009

Moores UCSD Cancer Center awarded $20 million for cancer research

Researchers led by Moores UCSD Cancer Center Director Dennis A. Carson, MD, professor of medicine, and Catriona Jamieson, MD, PhD, assistant professor of medicine and director of the Cancer Stem Cell Research Program at the Moores UCSD Cancer Center have been awarded $20 million over four years to develop novel drugs against leukemia stem cells.

28 Oct 2009

Study shows gene therapy can restore vision to children with Leber congenital amaurosis

Results from a breakthrough study, conducted by researchers at The Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind.

27 Oct 2009

Enzyme Mst3b is essential for regenerating damaged axons in live animal model, say researchers

Researchers at Children's Hospital Boston report that an enzyme known as Mst3b, previously identified in their lab, is essential for regenerating damaged axons (nerve fibers) in a live animal model, in both the peripheral and central nervous systems.

26 Oct 2009

Gene therapy may help improve congenital blindness in children

Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.

26 Oct 2009

Follow healthy lifestyle to overturn genetic predisposition to heart disease

When it comes to heart disease, lifestyle usually trumps genetics, says noted genetic researcher Dr. Robert Hegele, Heart and Stroke Foundation of Canada Lecturer at the Canadian Cardiovascular Congress 2009.

23 Oct 2009

Book reviews applications of genetic research in the fields of exercise science and sports medicine

This publication reviews past, current and future applications of genetic research in the fields of exercise science and sports medicine. It highlights ethical concerns, potential clinical applications and exploitation of genetic information. The authors, an interdisciplinary group of experts comprising clinicians, exercise scientists, human geneticists and other biological scientists, present an integrated and holistic understanding of the field to the reader.

21 Oct 2009

Encorium Group raises $1.6 million through private placement

Encorium Group, Inc., a full-service multinational contract research organization (CRO) that provides design, development, and management capabilities for clinical trials and patient registries to many of the world's leading pharmaceutical companies, today announced that it has completed a private placement of 3,937,500 shares of its common stock with a private investor for an aggregate purchase price of $1,575,000, or $.40 per share.

19 Oct 2009

Cardium Therapeutics reports positive findings from Excellarate Phase 1/2 clinical study

Cardium Therapeutics today announced the publication of positive findings from the open label multi-center Phase 1/2 clinical study of Excellarate(TM) (GAM501, Ad5PDGF-B / 2.6% collagen), its product candidate for the potential treatment of non-healing diabetic foot ulcers.

19 Oct 2009

Genetic mutation implicated in breast and ovarian cancers could also have a role in causing heart failure: Study

A genetic mutation implicated in breast and ovarian cancers could also have a role in causing heart failure, according to a new study reported at the 95th annual Clinical Congress of the American College of Surgeons.

15 Oct 2009

SCID infants who receive early bone marrow transplants are likely to have fewer complications

Mention the words "bubble boy" and many will recall David Vetter, the kid with big eyes and a thick thatch of dark hair who died 25 years ago after spending almost the entire 12 years of his life in a germ-free, plastic bubble. David was born with severe combined immune deficiency, or SCID, a condition that robbed him of an immune system.

14 Oct 2009

New UCL cancer research centre launched in UK

A new centre is launched in London today (Wednesday), joining a select group of Cancer Research UK Centres across the UK.

14 Oct 2009

SAFC Pharma expands contract manufacturing services

SAFC Pharma®, a business segment of SAFC®, a member of the Sigma-Aldrich® Group, today announced the completion of an addition to its Carlsbad, Calif., facility that expands the Company's contract manufacturing services for its late phase and commercial clients.

13 Oct 2009

Amsterdam Molecular Therapeutics' AMT-080 granted EMEA Orphan Drug Designation

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.

13 Oct 2009

New York Governor participates in roundtable discussion on stem cell research

Albert Einstein College of Medicine of Yeshiva University hosted a roundtable discussion on stem cell research with New York Governor David A. Paterson today. Allen M. Spiegel, M.D., the Marilyn and Stanley M. Katz Dean of Einstein, and eight stem cell researchers discussed advances in medical therapies and treatments that Einstein scientists have been investigating since receiving more than $14 million in State funding for stem cell research.

9 Oct 2009

Clinical trial of gene transfer therapy for advanced Parkinson's disease

Henry Ford West Bloomfield Hospital is embarking on a research study for advanced Parkinson's disease using a state-of-the-art treatment called gene transfer.

7 Oct 2009

Gene therapy to improve ailing heart's contractions of muscle cells

Scientists from the Universities of Michigan and Minnesota show in a research report published online in the FASEB Journal (http://www.fasebj.org) that gene therapy may be used to improve an ailing heart's ability to contract properly. In addition to showing gene therapy's potential for reversing the course of heart failure, it also offers a tantalizing glimpse of a day when "closed heart surgery" via gene therapy is as commonly prescribed as today's cocktail of drugs.

6 Oct 2009

Study points to subtle differences between boys and girls' attitudes to multiple drugs

New research by Weill Cornell Medical College researchers looks at the specific ways parents and peers influence teenagers to smoke, drink and use marijuana in combination.

3 Oct 2009

Effect of healthcare reform on treating cardiovascular disease patients

The Ninth Annual Controversies and Advances in Treatment of Cardiovascular Disease continuing medical education conference will feature several discussions and presentations on how healthcare reform could affect cardiovascular disease patients' diagnosis, treatment and outcome.

1 Oct 2009