Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

“This is the beginning of a new era of medicine,” said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania’s Perelman School of Medicine. He added that the method ...

The goal of the new therapy was “to be very precise” and “just deliver the gene that’s missing.” The result is a treatment that is safer and more effective with significantly fewer side ...

Genetics researcher Jonathan Rios talks about his research to test a gene therapy for a rare neurological disease in children called childhood-onset striatonigral degeneration, at Dallas Scottish ...

Gene therapy advancements necessitate improved delivery infrastructure and comprehensive data collection across age groups to address logistical complexities and policy gaps. Multidisciplinary ...

Get Instant Summarized Text (Gist) A novel gene therapy has been developed that alleviates symptoms of Dravet syndrome in mice without side effects. This therapy uses a two-step strategy ...

Yet on Tuesday, Sarepta shares sank as much as 25% after the company reported the death of a young man from acute liver failure following treatment with its gene therapy. The news was ...

HYDERABAD, March 20 (Reuters) - Indian vaccine maker Bharat Biotech said on Thursday it had invested $75 million in its first cell and gene therapy (CGT) facility in the southern Indian state of ...