A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...
Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...
The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...
As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. The series C was led by Arch ...
Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...
Ocugen faces a packed 2026-27 catalyst window with filings, interim data and Phase III readouts across three one-time retinal gene therapies.
The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical milestone.
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Harvard couple wins $3M 'Oscar of science' for first gene therapy to reverse childhood blindness
A married couple of scientists has won the 2026 Breakthrough Prize in Life Sciences for creating the world's first gene therapy to restore sight in patients with a rare form of inherited blindness.
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