Genetics

Gene therapy startup to increase AAV gene therapy efficiency

Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project ...

Oncology & Cancer

Girl aged 5 dies in US during French gene therapy trial

A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said Thursday.

Diseases, Conditions, Syndromes

Cure found for rare form of inflammatory bowel disease

A rare genetic condition which causes inflammatory bowel disease can be successfully treated by bone marrow transplant, according to University of Manchester and Manchester University NHS Foundation Trust researchers.

Medical research

Drugging the undruggable: A treatment path for muscular dystrophy

Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered 'undruggable.' ...

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