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A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.
Christensen CL, Choy FYM
Abstract:
...In this review, the overall utility of the CRISPR/Cas9 gene editing technique for treatment of genetic diseases, the potential for the treatment of LSDs and methods currently employed to increase the efficiency of this re-engineered biological system will be discussed.
PMID: 28933359
Free Full-Text: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5456334/
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