CRISPR/Cas9-Mediated Three Nucleotide Insertion Corrects a Deletion Mutation in MRP1/ABCC1 and Restores Its Proper Folding and Function

CRISPR/Cas9-Mediated Three Nucleotide Insertion Corrects a Deletion Mutation in MRP1/ABCC1 and Restores Its Proper Folding and Function

A three-nucleotide deletion in cystic fibrosis transmembrane conductance regulator/ATP-binding cassette transporter C7 (CFTR/ABCC7) resulting in the absence of phenylalanine at 508 leads to mis-fold of the mutated protein and causes cystic fibrosis. We have used a comparable three-nucleotide deletio...

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Bibliographic Details
Main Authors: Qinqin Xu, Yue-xian Hou, Xiu-bao Chang
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MRP1/ABCC1-Δ
F728
CRISPR-Cas9
HDR
NHEJ
donor DNA
Δ
F728 target DNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301750

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http://www.sciencedirect.com/science/article/pii/S2162253117301750

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