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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Detalles Bibliográficos
Autores principales: Xu, Qinqin, Hou, Yue-xian, Chang, Xiu-bao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443964/
https://www.ncbi.nlm.nih.gov/pubmed/28624219
http://dx.doi.org/10.1016/j.omtn.2017.05.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5443964/
https://www.ncbi.nlm.nih.gov/pubmed/28624219
http://dx.doi.org/10.1016/j.omtn.2017.05.005

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