Article
In Vivo Base Editing Partially Rescues Bone Dysplasia in a Mouse Model of Hutchinson-Gilford Progeria Syndrome.
Aging cell - 1 Sept 2026
Cabral Wayne A, Grenko Caleb M, Yeritsyan Diana, Beeram Indeevar, Laiwala Shubham, Johnson Lukas R, Tavarez Urraca L, Yan Tingfen, Wincovitch Stephen M, Koblan Luke W, Gao Xin D, An Meirui, Narisu Narisu, Biesecker Leslie G, Liu David R, Nazarian Ara, Collins Francis S, Erdos Michael R
Abstract excerpt
Hutchinson-Gilford progeria syndrome (HGPS) is a premature aging disorder affecting tissues of mesenchymal origin. Most patients harbor a c.1824C>T/p.G608= variant, commonly described as G608G, in exon 11 of LMNA that leads to aberrant splicing and production of the toxic progerin protein. In addition to cardiovascular, dermal, and adipose tissue deterioration, HGPS mouse models also develop progressive bone...
Read the complete abstract on PubMedTopics
- Animals
- Progeria
- Disease Models, Animal
- Mice
- Gene Editing