Article

Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells

2018-08-22

Abstract excerpt

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. High frequencies of reproducible targeted integration of a wild-type cDNA into the endogenous start codon of a gene in LT-HSCs could provide a robust genome editing approach to cure genetic diseases in which patients have different mutations throughout the gene....

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Literature Corpus work: 8a382112-53c5-5718-9def-41c46c986e86
DOI: 10.1101/397463

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