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.

Genetic basis unknown in many children with mental retardation and developmental delays

Researchers from four laboratories that perform diagnostic genetic testing of chromosome abnormalities in individuals with unexplained physical and developmental disabilities recently identified a previously unrecognized genetic disorder.

10 Mar 2010

AMT initiates dosing in Phase I/II gene therapy clinical trial for hemophilia B

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the first patient has been dosed in the Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B, a seriously debilitating and potentially lethal disease.

10 Mar 2010

New study may lead to expansion of drug arsenal used to fight HIV

New findings from a Universit- de Montr-al and the Vaccine and Gene Therapy Institute of Florida (VGTI) study, in collaboration with scientists from the NIH and the McGill University Health Center, may soon lead to an expansion of the drug arsenal used to fight HIV.

9 Mar 2010

Endothelial cells can produce copious amounts of adult stem cells: Study

In a leap toward making stem cell therapy widely available, researchers at the Ansary Stem Cell Institute at Weill Cornell Medical College have discovered that endothelial cells, the most basic building blocks of the vascular system, produce growth factors that can grow copious amounts of adult stem cells and their progeny over the course of weeks.

5 Mar 2010

Gene therapy for LCA clears another hurdle: Study

Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection.

4 Mar 2010

Robotic surgery does not compromise clinical outcomes for prostate cancer surgery

Robotic surgical technology with its three-dimensional, high-definition view gives surgeons the sensation of touch, even as they operate from a remote console. A new study describes the phenomenon, called intersensory integration, and reports that surgical outcomes for prostate cancer surgery using minimally invasive robotic technology compare favorably with traditional invasive surgery.

3 Mar 2010

Greffex Group signs Strategic Alliance Framework Agreement with UPMC and ISMETT

The Greffex Group of regenerative medicine companies announces the signing of a Strategic Alliance Framework Agreement with UPMC and ISMETT, Italy.

2 Mar 2010

BioSante Pharmaceuticals announces receipt of two US patents directed to 2A/Furin technology

BioSante Pharmaceuticals, Inc. today announced the receipt of two US patents directed to its 2A/Furin technology for expressing proteins. The first issued US patent, US Pat. No., 7,632,059, covers methods for manufacturing recombinant antibodies.

2 Mar 2010

Reversing protein deficiency through gene therapy can improve SMA

Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy, new research shows.

1 Mar 2010

Electronic prescribing decreases prescription errors

Should doctors around the country use e-prescribing to decrease prescription errors? A study led by physician-scientists from Weill Cornell Medical College found that health care providers using an electronic system to write prescriptions were seven times less likely to make errors than those writing their prescriptions by hand. The study appears today in the online edition of the Journal of General Internal Medicine.

27 Feb 2010

A surgical cure for diabetic and obese patients

Dr. Francesco Rubino, chief of gastrointestinal metabolic surgery at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, is now enrolling overweight and mildly obese patients -- those with a body mass index (BMI) of 28 to 35 -- in a study of gastric bypass surgery aimed at reversing Type 2 diabetes.

25 Feb 2010

Scientists one step closer to innovative drug delivery

A team of scientists led by Dr Simon Richardson at the University of Greenwich has got a step closer to one of the holy grails of drug delivery.

25 Feb 2010

Mouse study provides new way to test novel therapies for liver infections

How do you study-and try to cure in the laboratory-an infection that only humans can get? A team led by Salk Institute researchers does it by generating a mouse with an almost completely human liver.

24 Feb 2010

AMT announces full year 2009 results; reports modest decrease in operating expenses

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2009.

24 Feb 2010

New Barts Cancer Research UK Centre launched

A new cancer centre, launched today, puts London at the forefront of cancer research.

23 Feb 2010

WCMC-Q students receive new round of UREP funds totaling $280,000

Twenty-eight WCMC-Q students have received grants totaling $280,000 in the latest, seventh, round of funding from the Undergraduate Research Experience Program (UREP) to conduct advanced research projects with faculty members.

22 Feb 2010

Special issue reviews current state of science in fetal therapy

Repairing birth defects in the womb. Inserting a tiny laser into the mother's uterus to seal off an abnormal blood flow and save fetal twins. Advancing the science that may allow doctors to deliver cells or DNA to treat sickle cell anemia and other genetic diseases before birth.

17 Feb 2010

Clive Svendsen to receive American Academy of Neurology Sheila Essey Award for his research on ALS

Clive Svendsen, Ph.D., director of the Cedars-Sinai Regenerative Medicine Institute, is receiving the American Academy of Neurology Sheila Essey Award for his research on ALS (amyothrophic lateral sclerosis).

16 Feb 2010

Children's Hospital of Philadelphia awarded two grants to research into cellular therapies for blood disorders

The new decade may herald an era of cell therapy -- treating human diseases by delivering highly specific beneficial cells. In the wake of an NIH decision late last year permitting federally funded researchers to use new lines of human embryonic stem cells, the door has opened more widely to stem cell research.

16 Feb 2010

Better and standardized animal models needed for musculoskeletal regenerative medicine

Clinical testing and development of novel therapies based on advances in tissue engineering and regenerative medicine that will one day enable the repair and replacement of diseased or damaged human muscle, bone, tendons, and ligaments depends on the availability of good animal models.

12 Feb 2010