Chapter title |
Nuclease-Mediated Double-Strand Break (DSB) Enhancement of Small Fragment Homologous Recombination (SFHR) Gene Modification in Human-Induced Pluripotent Stem Cells (hiPSCs).
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Chapter number | 18 |
Book title |
Gene Correction
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Published in |
Methods in molecular biology, February 2014
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DOI | 10.1007/978-1-62703-761-7_18 |
Pubmed ID | |
Book ISBNs |
978-1-62703-760-0, 978-1-62703-761-7
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Authors |
Sargent RG, Suzuki S, Gruenert DC, R. Geoffrey Sargent, Shingo Suzuki, Dieter C. Gruenert, Sargent, R. Geoffrey, Suzuki, Shingo, Gruenert, Dieter C. |
Abstract |
Recent developments in methods to specifically modify genomic DNA using sequence-specific endonucleases and donor DNA have opened the door to a new therapeutic paradigm for cell and gene therapy of inherited diseases. Sequence-specific endonucleases, in particular transcription activator-like (TAL) effector nucleases (TALENs), have been coupled with polynucleotide small/short DNA fragments (SDFs) to correct the most common mutation in the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene, a 3-base-pair deletion at codon 508 (delF508), in induced pluripotent stem (iPS) cells. The studies presented here describe the generation of candidate TALENs and their co-transfection with wild-type (wt) CFTR-SDFs into CF-iPS cells homozygous for the delF508 mutation. Using an allele-specific PCR (AS-PCR)-based cyclic enrichment protocol, clonal populations of corrected CF-iPS cells were isolated and expanded. |
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