Previous efforts to circumvent the problem of the size limitation imposed by the AAV vector have focused mainly on altering the candidate genes into ‘mini-expression cassettes’ suitable for ...

This research evaluates the effects of various gene therapy viral vectors on RPE cells to determine efficacy for therapeutic ...

Delivering very large genes to cells can pose a serious challenge, so scientists developed a new method. | Genetics And Genomics ...

A patient has died after receiving the high dose of Neurogene’s Rett syndro | A patient has died after receiving the high ...

OXB to host a free webinar on accelerating development of viral vectors through automated manufacturing and analytical developmentOxford, UK – 20 ...

Ascend Advanced Therapies, which has been on an expansion tear since debuting three years ago, inked a deal with EW ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...

In neuromuscular diseases, the most widely used vector for transporting genetic material is the natural adeno-associated virus (AAV). However, a large proportion of injected vectors do not reach ...

As infection with the vector is not harmful and AAV cannot reproduce on its own, AAV-mediated gene therapy is gaining the most attention among them. The AAV viral vectors, as well as messenger ...