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37. Towards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 Mutations

Year: 2016
Type: article
Abstract: The scope of genetic engineering of hematopoietic stem/progenitor cells (HSPC) has broadened from gene replacement to targeted genome editing using a choice of artificial nucleases, which enables precise modifications of endogenous genes. We recently showed that gene editing in the primitive HSPC is constrained by gene transfer efficiency and limited proficiency of homology directed DNA repair. By combining Integrase Defective Lentiviral Vectors (IDLV) for donor template delivery and mRNA transfection for nucleases expression and tailoring culture conditions, we overcame in part these barriers and provided evidence of increased targeted integration (TI) in human HSPC.
DOI: 10.1016/s1525-0016(16)32846-5
Authors: Pietro Genovese, Giulia Schiroli, Anthony Conway, Samuele Ferrari, Dmitry Y. Guschin, Tiziano Di Tomaso, Luisa Albano, Virna Marin, Erica Giuliani, Philip D. Gregory, Michael C. Holmes, Bernhard Gentner, Chiara Bovolenta, Gregory J. Cost, Angelo Lombardo, Luigi Naldini
Language: English
Subfield: Genetics
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2563740134
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