Novel HDAd/EBV Reprogramming Vector and Highly Efficient Ad/CRISPR-Cas Sickle Cell Disease Gene Correction

CRISPR/Cas enhanced correction of the sickle cell disease (SCD) genetic defect in patient-specific induced Pluripotent Stem Cells (iPSCs) provides a potential gene therapy for this debilitating disease. An advantage of this approach is that corrected iPSCs that are free of off-target modifications c...

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Veröffentlicht in:Sci Rep
Hauptverfasser: Li, Chao, Ding, Lei, Sun, Chiao-Wang, Wu, Li-Chen, Zhou, Dewang, Pawlik, Kevin M., Khodadadi-Jamayran, Alireza, Westin, Erik, Goldman, Frederick D., Townes, Tim M.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2016
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4961958/
https://ncbi.nlm.nih.gov/pubmed/27460639
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep30422
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