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Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes

CRISPR/Cas9 systems have been used in a wide variety of biological studies; however, the large size of CRISPR/Cas9 presents challenges in packaging it within adeno-associated viruses (AAVs) for clinical applications. We identified a two-cassette system expressing pieces of the S. pyogenes Cas9 (SpCa...

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Detalles Bibliográficos
Autores principales: Fine, Eli J., Appleton, Caleb M., White, Douglas E., Brown, Matthew T., Deshmukh, Harshavardhan, Kemp, Melissa L., Bao, Gang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4486982/
https://www.ncbi.nlm.nih.gov/pubmed/26126518
http://dx.doi.org/10.1038/srep10777