CRISPR/Cas9 PIG-A gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs
Year: 2019
Type: article
Abstract: Recent advances in gene editing technologies using CRISPR/ Cas9 allow precise genome editing at a site of interest and have accelerated human disease modeling and the development of corrective gene therapies for various genetic disorders.1,2 We adapted CRISPR/Cas9 editing of rhesus macaque (RM) hematopoietic stem and progenitor cells (HSPCs) to create the first engineered large animal model of a hematologic disease based on close phylogenetic/functional similarity of RM to human HSPCs.
Source: Blood
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: bronze
OpenAlex ID: W2938625561