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

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

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 pr...

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Bibliographic Details
Main Authors: Chloe L. Christensen, Francis Y. M. Choy
Format: Article
Language:English
Published: MDPI AG 2017-02-01
Series:Diseases
Subjects:
CRISPR-Cas9
gene editing
lysosomal storage disease
induced pluripotent stem cells
genetic disease
Online Access:http://www.mdpi.com/2079-9721/5/1/6

Internet

http://www.mdpi.com/2079-9721/5/1/6

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