
NCBI . NLM . NIH . GOV {
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Title[redir]:
Homology-driven genome editing in hematopoietic stem and progenitor cells using zinc finger nuclease mRNA and AAV6 donors - PMC
Description:
Genome editing with targeted nucleases and DNA donor templates homologous to the break site has proven challenging in human hematopoietic stem and progenitor cells (HSPCs), and particularly in the most primitive, long-term repopulating cell ...
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Keywords {🔍}
cells, doi, hspcs, aav, ccr, pubmed, google, scholar, vectors, gfp, genome, human, editing, blood, cell, mice, gene, pmc, zfn, article, mrna, levels, donor, fetal, fig, free, mobilized, aavs, expression, stem, treated, population, insertion, pcr, liver, vector, locus, dna, supplemental, hematopoietic, zfns, ccrgfp, nat, analysis, data, shown, nucleases, sitespecific, flow, sequences,
Topics {✒️}
wiskott-aldrich syndrome—long-term efficacy 2a+2b+2c+2d+2e+2f long-term multi-lineage production cfu-granulocyte/erythroid/macrophage/megakaryocyte enhancing zinc-finger-nuclease activity long-term scid-repopulating cells semi-solid methylcellulose-based medium rna-guided crispr cas9 long-term repopulating activity22 long-term repopulating hscs46 ongoing multi-lineage hematopoiesis44 nhej-mediated end-capture events39–41 20ng/ml g-csf long-term-repopulating hscs primitive cd34+cd133+cd90+ subset long-term multilineage engraftment cd34+cd133+cd90+ cell population γ-globin repressor bcl11a15 custom-written computer script cg-prkcdscid il2rgtm1wjl/szj cd34+thy− 1+lin− subpopulation burst forming unit-erythroid retrovirus-mediated gene therapy achieving site-specific insertion long-term engraftment potential cftr-δf508 heterozygous pigs crispr/cas-based methods confer long-lasting effects 5μg/ml propidium iodide pmc beta search cmv-gfp reporter vectors zinc-finger nuclease specificity sickle-cell disease mutation integration-defective lentiviral vectors include zinc-finger nucleases site-specific genome editing hspc-tropic serotype aav6 site-specific integration event fetal liver–derived hspcs magnetic-activated cell sorting erythroid lineage-restricted expression programmable site-specific nucleases hdr-mediated genome editing fetal liver–derived cells inverted terminal repeats fast gapped-read alignment site-specific rflp insertions virus–mediated gene targeting improved dna-cleavage specificity site-specific gfp insertion
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