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Paper Details

Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.
Gene Ther
32
2021
1-13 or 2-13 cDNAs, 13, 53BP1, CRISPR, CYBB, CYBB cDNA, CYBB exon, CYBB exon 1 or, CYBB gene, Cas9, HSPCs, X-CGD, X-linked chronic granulomatous disease, endogenous, exon 1-13 cDNA, exon 2 sites, exon 2-13 cDNA, exon 2-13 corrected cells, exons, gp91phox, gp91phox protein, hematopoietic stem/progenitor cells, i53 mRNA, immunodeficiency, intron, intron 1, mice, oxygen, patient, phagocyte, phagocytes, regulatory elements, splice sites, target gene, woodchuck hepatitis virus, woodchuck hepatitis virus post-transcriptional regulatory element
Author NameAffiliation
Xiaolin WuLeidos Biomedical Research Inc.
Xiaolin WuLeidos Biomedical Research Inc.
Matthew H PorteusStanford University School of Medicine
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Release 1.2