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.

Researchers discover herpes virus-associated disease in monkeys

Researchers at Oregon Health & Science University have discovered a naturally occurring disease in monkeys that is very much like multiple sclerosis in humans - a discovery that could have a major impact on efforts to understand the cause of multiple sclerosis.

29 Jun 2011

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today released data showing that its gene therapy Glybera® (alipogene tiparvovec) significantly reduces the risk of pancreatitis in patients with Lipoprotein Lipase Deficiency (LPLD). Pancreatitis, or inflammation of the pancreas, is a major clinical symptom of LPLD.

29 Jun 2011

Genetic tools that enable cell type-specific delivery hold promise for combating incurable neurologic diseases

Novel tools and methods for delivering therapeutic genes to cells in the central nervous system hold great promise for the development of new treatments to combat incurable neurologic diseases. Five of the most exciting developments in this rapidly advancing field are presented in a series of articles in the June issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc..

29 Jun 2011

Circulation journal publishes Celladon's MYDICAR Phase 2 trial on advanced heart failure

Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, announces that the data from its completed Phase 2 CUPID clinical study of MYDICAR for the treatment of advanced heart failure is being published today online in Circulation, an American Heart Association journal.

28 Jun 2011

Flamel, Digna Biotech enter joint development agreement for multiple products

Flamel Technologies SA and Digna Biotech SL today announced that the two companies have entered into a joint development agreement for the pre-clinical and clinical development of multiple products.

27 Jun 2011

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice.

27 Jun 2011

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

New surprising details about flow of neurotransmitters between brain neurons

Two studies featuring research from Weill Cornell Medical College have uncovered surprising details about the complex process that leads to the flow of neurotransmitters between brain neurons -- a dance of chemical messages so delicate that missteps often lead to neurological dysfunction.

23 Jun 2011

Telaprevir drug shows promise in curing hepatitis C infection

The drug telaprevir (Incivek) provides a dramatic improvement in the treatment of the most common form of hepatitis C infection, says an international team of investigators led by Dr. Ira M. Jacobson of NewYork-Presbyterian Hospital/Weill Cornell Medical Center.

23 Jun 2011

Oxford BioMedica reports RetinoStat progress in Phase I study against wet AMD

Oxford BioMedica plc, a leading gene therapy company, today announces that the first dose level of RetinoStat is safe and well-tolerated at one month following treatment.

22 Jun 2011

Benitec’s pain program validated by independent study

Benitec Ltd (ASX:BLT), a world leader in RNA-based gene silencing for human therapeutics, announced that China-based researchers have proved the concept behind Benitec's in house neuropathic pain program.

20 Jun 2011

Researchers use DNA library to create potential cancer vaccine

University of Leeds researchers, funded by Cancer Research UK, have used a library of DNA to create a vaccine that could be used to treat cancer, according to a study published in Nature Medicine.

20 Jun 2011

Varicocele surgery may increase testosterone production

17 Jun 2011

Cedars-Sinai receives National Eye Institute grant to develop gene therapy for corneal disease

Cedars-Sinai stem cell researchers investigating ways to prevent eye problems in diabetic patients have been awarded a $3 million grant from the National Eye Institute to develop gene therapy in corneal stem cells to alleviate damage to corneas that can cause vision loss.

17 Jun 2011

Low testosterone linked to varicoceles

As many as 15 percent of men have varicoceles, masses of enlarged and dilated veins in the testicles. There is new evidence that varicoceles, long known to be a cause of male infertility, interfere with the production of testosterone -- a crucial hormone to maintaining men's health. There is good news too: Microsurgery can increase testosterone levels in these men. Results of new research by physician-scientists at NewYork-Presbyterian Hospital/Weill Cornell Medical Center are in a recent edition of BJU International.

16 Jun 2011

High number of adverse patient safety events occur in doctors' offices

However, a high number of adverse events, including major injury and even death, occur in private physician offices and outpatient clinics as well. In a new study -- the first of its kind -- researchers at Weill Cornell Medical College found that the number and magnitude of events resulting from medical errors is surprisingly similar inside and outside hospital walls.

15 Jun 2011

Newborn neurons help create emotional context of memory

Fear burns memories into our brain, and new research by University of California, Berkeley, neuroscientists explains how.

15 Jun 2011

Individuals with chronic vascular disease can benefit from echo-mediated gene therapy

Researchers today announced the results of a study that show echocardiography can improve the success rate of gene therapy and increase growth, function and sustainability of regenerated vessels.

14 Jun 2011

Neurologix reports new results from NLX-P101 Phase 2 trial against Parkinson's disease

Neurologix, Inc. announced today several new results from the Company's successful Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD) as part of a comprehensive presentation of study findings to the National Institutes of Health (NIH) Recombinant DNA Advisory Committee.

9 Jun 2011

ImaginAb, Oxford BioMedicaplc enter research deal to develop in vivo diagnostic imaging agent

Oxford BioMedicaplc, a leading gene therapy company, today announces that it has signed a research collaboration agreement with ImaginAb, Inc., a biotechnology company specialising in the development of engineered antibody fragments for diagnostic imaging and novel therapeutic applications, to engineer an in vivo diagnostic imaging agent using an antibody targeting the Company's proprietary 5T4 tumour antigen.

7 Jun 2011

Gene Therapy News and Research

Further Reading

Researchers discover herpes virus-associated disease in monkeys

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

Genetic tools that enable cell type-specific delivery hold promise for combating incurable neurologic diseases

Circulation journal publishes Celladon's MYDICAR Phase 2 trial on advanced heart failure

Flamel, Digna Biotech enter joint development agreement for multiple products

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

New surprising details about flow of neurotransmitters between brain neurons

Telaprevir drug shows promise in curing hepatitis C infection

Oxford BioMedica reports RetinoStat progress in Phase I study against wet AMD

Benitec’s pain program validated by independent study

Researchers use DNA library to create potential cancer vaccine

Varicocele surgery may increase testosterone production

Cedars-Sinai receives National Eye Institute grant to develop gene therapy for corneal disease

Low testosterone linked to varicoceles

High number of adverse patient safety events occur in doctors' offices

Newborn neurons help create emotional context of memory

Individuals with chronic vascular disease can benefit from echo-mediated gene therapy

Neurologix reports new results from NLX-P101 Phase 2 trial against Parkinson's disease

ImaginAb, Oxford BioMedicaplc enter research deal to develop in vivo diagnostic imaging agent

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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