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Diseases 2017, 5(1), 6; doi:10.3390/diseases5010006

A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells

Department of Biology, Centre for Biomedical Research, University of Victoria, 3800 Finnerty Rd., Victoria, BC V8P 5C2, Canada
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Author to whom correspondence should be addressed.
Academic Editor: Jose A. Sanchez-Alcazar
Received: 1 December 2016 / Revised: 15 February 2017 / Accepted: 20 February 2017 / Published: 24 February 2017
(This article belongs to the Collection Lysosomal Storage Diseases)
View Full-Text   |   Download PDF [489 KB, uploaded 24 February 2017]   |  

Abstract

Ease of design, relatively low cost and a multitude of gene-altering capabilities have all led to the adoption of the sophisticated and yet simple gene editing system: clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9). The CRISPR/Cas9 system holds promise for the correction of deleterious mutations by taking advantage of the homology directed repair pathway and by supplying a correction template to the affected patient’s cells. Currently, this technique is being applied in vitro in human-induced pluripotent stem cells (iPSCs) to correct a variety of severe genetic diseases, but has not as of yet been used in iPSCs derived from patients affected with a lysosomal storage disease (LSD). If adopted into clinical practice, corrected iPSCs derived from cells that originate from the patient themselves could be used for therapeutic amelioration of LSD symptoms without the risks associated with allogeneic stem cell transplantation. CRISPR/Cas9 editing in a patient’s cells would overcome the costly, lifelong process associated with currently available treatment methods, including enzyme replacement and substrate reduction therapies. 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. View Full-Text
Keywords: CRISPR-Cas9; gene editing; lysosomal storage disease; induced pluripotent stem cells; genetic disease CRISPR-Cas9; gene editing; lysosomal storage disease; induced pluripotent stem cells; genetic disease
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Christensen, C.L.; Choy, F.Y.M. A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells. Diseases 2017, 5, 6.

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