Accès ouvert
2026
article
OpenAlex
Tyler J. Brittain, Seongmin Jang, Gerard Michael Coughlin, Jonathan D. Hoang et autres
Developing effective vectors for gene therapy requires accurate on-target coverage while minimizing off-target transduction that can lead to adverse events. In mice, the engineered capsid PHP.eB shows enhanced brain transduction, while the further engineered CAP-B10 is also de-targeted from astrocytes and liver. …
us
(code pays fourni par la source)
Accès ouvert
2025
preprint
OpenAlex
Tyler J. Brittain, Seongmin Jang, Gerard Michael Coughlin, Bre’Anna H. Barcelona et autres
Abstract Crossing the blood-brain barrier while minimizing liver transduction is a key challenge in developing safe adeno-associated virus (AAV) vectors for treating brain disorders. In mice, the engineered capsid PHP.eB shows enhanced brain transduction, while the further engineered CAP-B10 is also de-targeted …
us
(code pays fourni par la source)
Accès ouvert
2025
article
OpenAlex
Gerard Michael Coughlin, Máté Borsos, Bre’Anna H. Barcelona, Nathan Appling et autres
Cell-type-specific regulatory elements such as enhancers can direct expression of recombinant adeno-associated viruses (AAVs) to specific cell types, but this approach is limited by the relatively small packaging capacity of AAVs. In this study, we used spatial genomics to show that transcriptional …
us
(code pays fourni par la source)
Accès ouvert
2023
preprint
OpenAlex
Gerard Michael Coughlin, Máté Borsos, Nathan Appling, Bre’Anna H. Barcelona et autres
Abstract Integrating cell type-specific regulatory elements (e.g. enhancers) with recombinant adeno-associated viruses (AAVs) can provide broad and efficient genetic access to specific cell types. However, the packaging capacity of AAVs restricts the size of both the enhancers and the cargo that can …
us
(code pays fourni par la source)