Cardiovascular Progerin Suppression and Lamin A Restoration Rescue Hutchinson-Gilford Progeria Syndrome.

ABSTRACT:

Background

Hutchinson-Gilford progeria syndrome (HGPS) is a rare disorder characterized by premature aging and death mainly because of myocardial infarction, stroke, or heart failure. The disease is provoked by progerin, a variant of lamin A expressed in most differentiated cells. Patients look healthy at birth, and symptoms typically emerge in the first or second year of life. Assessing the reversibility of progerin-induced damage and the relative contribution of specific cell types is critical to determining the potential benefits of late treatment and to developing new therapies.

Methods

We used CRISPR-Cas9 technology to generate Lmna^{HGPSrev/HGPSrev} (HGPSrev) mice engineered to ubiquitously express progerin while lacking lamin A and allowin

SUBMITTER: Sanchez-Lopez A 

PROVIDER: S-EPMC8614561 | biostudies-literature | 2021 Nov

REPOSITORIES: biostudies-literature