Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. The study, ...

Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes ...

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 ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in gene therapy using adeno-associated virus vectors (AAV), specifically ...

A new study describes the use of artificial intelligence (AI) in designing a new generation of capsids, the structures that envelop genetic material of adeno-associated viruses (AAV), to improve gene ...

Over the past few decades, there has been remarkable progress in genetic manipulation technologies, bringing us closer to the point where genes can be modified in vivo. Such tools would open up the ...

Researchers develop a nanosensing platform that can assess the quality of individual viral vector particles Viral vectors hold much potential for gene editing and gene therapy, but there is a pressing ...

Researchers reported few adverse events among pediatric X-linked retinoschisis patients who received a single, photoreceptor cell-targeted gene therapy dose.

Danish start-up Fuse Vectors has received $5.2 million in preseed financing to further develop their gene therapy technology. The company has developed a cell-free platform for producing ...