Ontology highlight
ABSTRACT:
SUBMITTER: Shapiro J
PROVIDER: S-EPMC7251314 | biostudies-literature | 2020 Jun
REPOSITORIES: biostudies-literature
Shapiro Jenny J Iancu Ortal O Jacobi Ashley M AM McNeill Matthew S MS Turk Rolf R Rettig Garrett R GR Amit Ido I Tovin-Recht Adi A Yakhini Zohar Z Behlke Mark A MA Hendel Ayal A
Molecular therapy. Methods & clinical development 20200504
Genome editing of human cluster of differentiation 34<sup>+</sup> (CD34<sup>+</sup>) hematopoietic stem and progenitor cells (HSPCs) holds great therapeutic potential. This study aimed to optimize on-target, <i>ex vivo</i> genome editing using the CRISPR-Cas9 system in CD34<sup>+</sup> HSPCs and to create a clear workflow for precise identification of off-target effects. Modified synthetic guide RNAs (gRNAs), either 2-part gRNA or single-guide RNA (sgRNA), were delivered to CD34<sup>+</sup> HSPC ...[more]