AAV vectors are well-suited to gene therapies for DMD because they are effective at ... US has been delayed in the US by an FDA request for potency, but is already underway elsewhere, including ...

potency testing, and biodistribution. Enhanced AAV Critical Quality Attributes (CQAs) Streamline the Transition from AAV Vector Design to Comprehensive Gene Therapy QC Characterization Guangping ...

A new study in the peer-reviewed journal Human Gene Therapy describes a machine learning (ML) model that can be used as a ...

the only FDA-approved gene therapy for DMD. Its maker, Sarepta Therapeutics, hasn’t ruled out the possibility that the treatment, which uses an adeno-associated virus vector to deliver its ...

Ashley Zukerman in a scene from In Vitro.Credit: Sydney Film Festival Cloning is the question under analysis in In Vitro, a low-budget Australian thriller set on a remote and rather spooky cattle ...

About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV ... or changes in the DMD gene that result in the ...

adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene ...