Medical research

Gene therapy cassettes improved for muscular dystrophy

Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment ...

Genetics

Making gene therapy delivery safer and more efficient

Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function, found researchers from the Perelman School of Medicine at the University of Pennsylvania ...

Neuroscience

New anti-ALS drug receives Orphan Drug designation

A new gene therapy for treating an inherited form of Amyotrophic Lateral Sclerosis has been given an Orphan Drug designation by the European Medicines Agency. The therapy is already in preclinical development at EPFL.

Genetics

A hiccup in gene therapy progress?

Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...

Genetics

AAV gene delivery vectors and cancer—The debate continues

Overwhelming evidence from the biomedical literature shows that adeno-associated virus 2 (AAV2), a viral vector often used to deliver therapeutic genes, is not associated with cancer and, in fact, may protect against cancer. ...

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