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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...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2015
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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 |