Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease

Ex vivo gene correction with CRISPR-Cas9 and a recombinant adeno-associated virus serotype 6 (rAAV6) in autologous hematopoietic stem/progenitor cells (HSPCs) to treat sickle cell disease (SCD) has now entered early-phase clinical investigation. To facilitate the progress of CRISPR-Cas9/rAAV6 genome...

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Detalles Bibliográficos
Autores principales: Xu, Liwen, Lahiri, Premanjali, Skowronski, Jason, Bhatia, Neehar, Lattanzi, Annalisa, Porteus, Matthew H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10447934/
https://www.ncbi.nlm.nih.gov/pubmed/37637384
http://dx.doi.org/10.1016/j.omtm.2023.07.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10447934/
https://www.ncbi.nlm.nih.gov/pubmed/37637384
http://dx.doi.org/10.1016/j.omtm.2023.07.009

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