According to a pair of NHL Insiders, the Devils and captain Nico Hischier are working toward a considerable contract ...

Adeno Associated Virus (AAV)-mediated gene expression in the brain is widely applied in the preclinical setting to investigate the therapeutic potential of specific molecular targets, characterize ...

The 1-year mortality rate of 20% in the study cohort suggests that holistic strategies where treatment is tailored to an individual’s frailty and disease activity, may help alleviate the elevated risk ...

Polyglutamine (PolyQ) diseases are caused by abnormal expansion of CAG repeats in the coding region of different genes. A shared therapeutic strategy against the gain-of-function toxicity of PolyQ ...

To design safe clinical trials, it is crucial to better understand and predict gene editing outcomes in preclinical studies. Bence György and collaborators have shown that adeno-associated viruses ...

Several quality attributes (QAs) must be monitored to ensure an effective and safe final product when developing adeno-associated virus (AAV) vectors as drug products. Thermal stability, aggregate ...

Patients with AAV are associated with an increased risk for severe infection compared with members of the general population.

After decades of research, gene therapy is finally on track to deliver solutions to myriad diseases once considered untreatable. More than 400 gene therapy trials are currently underway in the United ...

While there is no cure for ANCA-associated vasculitis (AAV), treatment is usually very effective at bringing it under control and preventing life-threatening complications. This group of rare ...

AskBio founder R. Jude Samulski talks about the evolution and future of AAV gene therapy following his company’s blockbuster acquisition by Bayer in 2020. R. Jude Samulski, PhD It is rare that a ...