According to the study, published in the journal Human Gene Therapy, co-injection of immunodominant peptides from the transgene product, along with AAV8, modulates the immune response at all AAV ...

A 1984 paper by Paul Hermonat and Nicholas Muzyczka offered the first demonstration that AAV might provide a safe and effective vehicle for transgene delivery, with helper adenovirus infection ...

Viral vectors expressing proneural genes can stimulate adult mammalian retinal glia to become immature neurons. These glia-derived neurons share properties with the bipolar, amacrine and retinal ...

Finally, AAV transduction was performed by transducing cerebral organoids (at 101 days) and cardiac organoids (at 11 days) with 1011 vg of eGFP transgene-bearing AAV5 WT as a negative control or ...

4 For instance, different AAV serotypes are better able to transduce particular ... aspects of the gene therapy product’s functionality including biodistribution, transgene expression, dose required ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock is sliding. The company shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne ...

A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which ...

confirming robust and efficient transduction of human photoreceptor-like cells by AAV vectors. The experiments demonstrated that a CAG-driven transgene transduced various cell types, whereas GRK1 ...

Elevidys is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy ... of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ...