Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS

Adeno-associated viruses derived from human hematopoietic stem cells (AAVHSCs) are naturally occurring AAVs. Fifteen AAVHSCs have demonstrated broad biodistribution while displaying differences in transduction. We examine the structure-function relationships of these natural amino acid variations on...

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Detalles Bibliográficos
Autores principales: Smith, Laura J., Schulman, Lindsay A., Smith, Samantha, Van Lieshout, Laura, Barnes, Carmen M., Behmoiras, Liana, Scarpitti, Meghan, Kivaa, Monicah, Duong, Khanh L., Benard, Ludo O., Ellsworth, Jeff L., Avila, Nancy, Faulkner, Deiby, Hayes, April, Lotterhand, Jason, Rivas, Jose Israel, Sengooba, Arnold V., Tzianabos, Alec, Seymour, Albert B., Francone, Omar L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9287613/
https://www.ncbi.nlm.nih.gov/pubmed/35859693
http://dx.doi.org/10.1016/j.omtm.2022.06.013

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