Ontology highlight
ABSTRACT:
SUBMITTER: Wang F
PROVIDER: S-EPMC7647984 | biostudies-literature | 2020 Nov
REPOSITORIES: biostudies-literature
Protein & cell 20200729 11
Many human genetic diseases, including Hutchinson-Gilford progeria syndrome (HGPS), are caused by single point mutations. HGPS is a rare disorder that causes premature aging and is usually caused by a de novo point mutation in the LMNA gene. Base editors (BEs) composed of a cytidine deaminase fused to CRISPR/Cas9 nickase are highly efficient at inducing C to T base conversions in a programmable manner and can be used to generate animal disease models with single amino-acid substitutions. Here, w ...[more]