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
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
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