In general, it should be an interesting goal for future work to compare the lead candidates from these and other studies side-by-side in genuine or humanized mouse livers in the presence of anti-AAV ...

However, questions have recently been raised about the accuracy of studies claiming efficient glia-to-neuron reprogramming in retina that were conducted using GFAP mini promoter-driven ...

“exhibits potent editing capabilities across various cell systems and tissues in vivo when administered via adeno-associated viral (AAV) vectors.” When targeting the PCSK9 gene in mouse liver ...

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He reports, “This is an RNA-based screening platform that has allowed us to create multiple families of novel, IV-delivered AAV capsids demonstrating robust penetration of the BBB. Our capsids ...

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Adeno-associated virus 2 (AAV)-based vectors have gained attention as a potentially useful alternative to the more commonly used retroviral and adenoviral vectors for human gene therapy.

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