Review paper

CRISPR/Cas in iPSCs from sphingolipidoses patients

Volume: 126, Issue: 2, Pages: S101 - S102
Published: Feb 1, 2019
Abstract
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) were found as an immune adaptive mechanism in bacteria and quickly were applied to various fields as a promising tool for gene editing. Lysosomal storage diseases (LSDs) are a group of metabolic disorders caused by defects in lysosomal proteins leading to accumulation of undigested macromolecules within the cells. The lack of good in vitro models hinders research of the...
Paper Details
Title
CRISPR/Cas in iPSCs from sphingolipidoses patients
Published Date
Feb 1, 2019
Volume
126
Issue
2
Pages
S101 - S102
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