Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo

Amy L. Ryan

doi:10.1016/j.stem.2020.03.012

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Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo

Journal article

Open access

Peer reviewed

Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo

Amy L. Ryan

Cell stem cell, Vol.26(4), pp.476-478

04/02/2020

DOI: 10.1016/j.stem.2020.03.012

PMID: 32243806

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Abstract

Gene correction presents one of few options for a cure for all patients with cystic fibrosis. This commentary discusses new applications of CRISPR-based gene editing technology with increased efficiency and specificity to correct the cystic fibrosis transmembrane regulator (CFTR) function in patient-specific primary epithelial cells.

Cell & Tissue Engineering

Cell Biology

Life Sciences & Biomedicine

Science & Technology

Details

Title: Subtitle: Correcting CFTR: New Gene Editing Strategies for Rescuing CFTR Function Ex Vivo
Creators: Amy L. Ryan - Hastings Center
Resource Type: Journal article
Publication Details: Cell stem cell, Vol.26(4), pp.476-478
DOI: 10.1016/j.stem.2020.03.012
PMID: 32243806
NLM abbreviation: Cell Stem Cell
ISSN: 1934-5909
eISSN: 1875-9777
Publisher: Elsevier
Number of pages: 3
Grant note: DOI: 10.13039/100000897, name: Cystic Fibrosis Foundation
Language: English
Date published: 04/02/2020
Academic Unit: Anatomy and Cell Biology
Record Identifier: 9984284457702771

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