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 ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

using an AAV vector encoding human leptin (AAVhOB) was assessed. As in the case of the studies involving AAVGFP, virus (3–4 × 10 10 c.f.u./ml) was injected into the lateral ventricle (IV ...

First generation vectors may be suitable for applications where transient, high level activity is sufficient, and where immune responses to the vector or ... into the adenovirus backbone plasmid, at ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

Biotech stock Neurogene collapsed Tuesday after a patient experienced a serious side effect in the company's Rett syndrome ...

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

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 ...

KEBILIDI is authorized to be administered using the SmartFlow Neuro Cannula. KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...