In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice

LW Koblan, MR Erdos, C Wilson, WA Cabral, JM Levy… - Nature, 2021 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS or progeria) is typically caused by a
dominant-negative C• G-to-T• A mutation (c. 1824 C> T; p. G608G) in LMNA, the gene that …

Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome

P Scaffidi, T Misteli - Nature medicine, 2005 - nature.com
Hutchinson-Gilford progeria syndrome (HGPS) is a childhood premature aging disease
caused by a spontaneous point mutation in lamin A (encoded by LMNA), one of the major …

A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome

MR Erdos, WA Cabral, UL Tavarez, K Cao… - Nature medicine, 2021 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS) is a rare accelerated aging disorder
characterized by premature death from myocardial infarction or stroke. It is caused by de …

Recurrent de novo point mutations in lamin A cause Hutchinson–Gilford progeria syndrome

M Eriksson, WT Brown, LB Gordon, MW Glynn, J Singer… - Nature, 2003 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS) is a rare genetic disorder characterized by
features reminiscent of marked premature ageing,. Here, we present evidence of mutations …

Generation of a Hutchinson–Gilford progeria syndrome monkey model by base editing

F Wang, W Zhang, Q Yang, Y Kang, Y Fan, J Wei… - Protein & …, 2020 - academic.oup.com
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 …

Rapamycin reverses cellular phenotypes and enhances mutant protein clearance in Hutchinson-Gilford progeria syndrome cells

K Cao, JJ Graziotto, CD Blair, JR Mazzulli… - Science translational …, 2011 - science.org
Hutchinson-Gilford progeria syndrome (HGPS) is a lethal genetic disorder characterized by
premature aging. HGPS is most commonly caused by a de novo single-nucleotide …

Inhibiting farnesylation reverses the nuclear morphology defect in a HeLa cell model for Hutchinson-Gilford progeria syndrome

MP Mallampalli, G Huyer, P Bendale… - Proceedings of the …, 2005 - National Acad Sciences
Hutchinson-Gilford progeria syndrome (HGPS) is a devastating premature aging disease
resulting from a mutation in the LMNA gene, which encodes nuclear lamins A and C. Lamin …

Interruption of progerin–lamin A/C binding ameliorates Hutchinson-Gilford progeria syndrome phenotype

SJ Lee, YS Jung, MH Yoon, S Kang… - The Journal of …, 2016 - Am Soc Clin Investig
Hutchinson-Gilford progeria syndrome (HGPS) is a rare autosomal dominant genetic
disease that is caused by a silent mutation of the LMNA gene encoding lamins A and C …

Recapitulation of premature ageing with iPSCs from Hutchinson–Gilford progeria syndrome

GH Liu, BZ Barkho, S Ruiz, D Diep, J Qu, SL Yang… - Nature, 2011 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS) is a rare and fatal human premature ageing
disease,,,,, characterized by premature arteriosclerosis and degeneration of vascular …

Farnesyltransferase inhibitor treatment restores chromosome territory positions and active chromosome dynamics in Hutchinson-Gilford progeria syndrome cells

IS Mehta, CH Eskiw, HD Arican, IR Kill, JM Bridger - Genome biology, 2011 - Springer
Abstract Background Hutchinson-Gilford progeria syndrome (HGPS) is a premature ageing
syndrome that affects children leading to premature death, usually from heart infarction or …