Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome.

ABSTRACT: CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene.

SUBMITTER: Santiago-Fernandez O 

PROVIDER: S-EPMC6546610 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature