Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae.

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2020年12月8日 · We have developed an efficient system to deliver Cas9 into the adeno-associated virus integration site 1 (AAVS1) locus, known as a safe harbor, using lentivirus and AAV viral vectors, as a step...

2024年12月24日 · Adeno-associated virus (AAV) has been reported to integrate in a site-specific manner into chromosome 19 (a site designated AAVS1), a phenomenon that could be exploited for ex vivo targeted gene therapy.

In this study we investigated the latent phase of serotype 2 AAV, focusing on three areas: AAV infection, rescue, and integration efficiency as a function of viral multiplicity of infection (MOI); efficiency of site-specific integration; and disruption of the AAVS1 locus.

2024年4月3日 · Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene...

2024年10月28日 · proclivity for adeno-associated virus integration at the AAVS1 site in chromosome 19

2024年10月1日 · AAVs hold great promise in the treatment of genetic diseases, but their clinical use has been limited by off-target transduction and efficiency. Here, we report a strategy to construct NACs by conjugating a nanobody or scFv to an rAAV capsid site, specifically via biorthogonal click chemistry and a spy-spycatcher reaction.

AAV is a non-pathogenic human parvovirus which is commonly isolated from the respiratory tract of humans (Figure 1A). AAV’s life cycle includes a unique mechanism for persistence in human cells by means of site-specific integration into a region of …

5 天之前 · The full length base editor and the split-573BE were tested as the positive control. Meanwhile, we also constructed the dual AAV split-A3ABE3.9max with the 511-split site. The editing efficiency from six target sites (RP11, NONO site 3, ZNF410 site 9, HDAC5, HEK1 and HERC5) were similar to that of the full-length base editors (Figure 2 B ...