Advances in Gene Therapy for Human Genetic Diseases Based on Crispr/Cas9 Gene Editing Technology
Journal Title: Genetic Disease Study - Year 2018, Vol 2, Issue 1
Abstract
The CRISPR/Cas9 system provides a powerful technique for targeted gene editing. Using sequence-specific sgRNA guidance, the CRISPR/Cas9 system can accurately introduce double-stranded incisions into the exact location of the target DNA. At present, a large number of CRISSR/Cas9 gene editing studies involving multiple species in vivo and in vitro have fully demonstrated the tremendous potential of this technology, which brings hope for disease treatment research and clinical application based on this technology. This review will summarize recent advances in the use of CRISPR/Cas9 gene editing techniques in the treatment of human genetic diseases. Progress in pre clinical research.
Authors and Affiliations
Delia Gaskell, Mosleh Abualhaj
Keywords
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