Adeno-associated viruses (AAV) have emerged as pivotal delivery vehicles for gene therapy due to their overall safety (less immunogenic), efficiency (broad tropism), and long-term gene expression.

Amid a reckoning for the gene therapy field, Vertex Pharmaceuticals has joined a growing list of companies paring back their research efforts around the adeno-associated virus (AAV) vectors used ...

“Cox was my No. 10 center at this time last week. When I update my Big Board this week, he might be in the C6 or C7 range,” Kiper wrote. “And he might have put himself in the fifth-round mix.

In addition, first-generation CRISPR systems (Cas9 and Cas12a) are too large for efficient in vivo delivery via a single adeno-associated viral (AAV) vector. Now, new preclinical research presents ...

March 2020 seems a very long time ago, but it was start of probably the biggest world event any of us will live through. Billions of people were effectively forced to stay home for months at a ...

EGFP was expressed in neurons in the hippocampi of 7-month-old mice injected with AAV-EGFP-sTREM2 or AAV-EGFP four months previously. It was reported that levels of expression of TREM2 in the ...

However, two large companion studies in non-human primates indicate that vector integrations in primate liver following AAV gene therapy may be an important mechanism for achieving durable ...

In AAV-based gene therapy, there is a crucial need for accurate and precise AAV titer. There is an urgent need for standardized quantification methods for AAV titer purposes. Developing and ...

EULAR first wrote recommendations for small and medium vessel vasculitis in 2009; this was subsequently updated in 2016 with more of a focus on AAV. The new 2022 update includes substantial ...

AAV is a primary vasculitis classified as small vasculitis in ... C5b binds to the surface of the target cells, then C6, C7, C8, and C9 sequentially bind to C5b to form the membrane attack complex ...

Adeno-associated virus (AAV) vectors are a versatile and appealing gene therapy delivery platform, capable of targeting a wide range of cell types. However, AAV must be produced in a living system, ...