Ontology highlight
ABSTRACT:
SUBMITTER: Pavel-Dinu M
PROVIDER: S-EPMC6456568 | biostudies-literature | 2019 Apr
REPOSITORIES: biostudies-literature
Nature communications 20190409 1
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. Here we describe an approach for X-linked sSevere cCombined iImmunodeficiency (SCID-X1) using targeted integration of a cDNA into the endogenous start codon to functionally correct disease-causing mutations throughout the gene. Using a CRISPR-Cas9/AAV6 based strategy, we achieve up to 20% targeted integration frequencies in LT-HSCs. As measure ...[more]