CCR5 editing by Staphylococcus aureus Cas9 in human primary CD4⁺ T cells and hematopoietic stem/progenitor cells promotes HIV-1 resistance and CD4⁺ T cell enrichment in humanized mice.

ABSTRACT:

Background

The chemokine receptor CCR5, which belongs to the superfamily of G protein-coupled receptors, is the major co-receptor for HIV-1 entry. Individuals with a homozygous CCR5Δ32 mutation have a long lasting and increased resistance to HIV-1 infection. Therefore, CCR5 represents an optimal target for HIV-1/AIDS gene therapy. The CRISPR/Cas9 system has been developed as one of the most efficacious gene editing tools in mammalian cells and the small-sized version from Staphylococcus aureus (SaCas9) has an advantage of easier delivery compared to the most commonly used version from Streptococcus pyogenes Cas9 (SpCas9).

Results

Here, we demonstrated that CCR5 could be specifically and efficiently edited by CRISPR/SaCas9 together with two sgRNAs, which were identified throu

SUBMITTER: Xiao Q 

PROVIDER: S-EPMC6560749 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature