Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome

E Beyret, HK Liao, M Yamamoto… - Nature medicine, 2019 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS) is a rare lethal genetic disorder
characterized by symptoms reminiscent of accelerated aging. The major underlying genetic …

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

O Santiago-Fernández, FG Osorio, V Quesada… - Nature medicine, 2019 - nature.com
CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic
diseases. Among these, Hutchinson–Gilford progeria syndrome, caused by a point mutation …

Cardiovascular progerin suppression and lamin A restoration rescue Hutchinson-Gilford progeria syndrome

A Sánchez-López, C Espinós-Estévez… - Circulation, 2021 - Am Heart Assoc
Background: Hutchinson-Gilford progeria syndrome (HGPS) is a rare disorder characterized
by premature aging and death mainly because of myocardial infarction, stroke, or heart …

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 …

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 …

Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome

M Puttaraju, M Jackson, S Klein, A Shilo, CF Bennett… - Nature medicine, 2021 - nature.com
Hutchinson–Gilford progeria syndrome (HGPS) is a rare, invariably fatal childhood
premature aging disorder caused by a pre-messenger RNA (mRNA) splicing defect in the …

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 …

Targeting of NAT10 enhances healthspan in a mouse model of human accelerated aging syndrome

G Balmus, D Larrieu, AC Barros, C Collins… - Nature …, 2018 - nature.com
Abstract Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare, but devastating genetic
disease characterized by segmental premature aging, with cardiovascular disease being the …

Genetic reduction of mTOR extends lifespan in a mouse model of Hutchinson‐Gilford Progeria syndrome

WA Cabral, UL Tavarez, I Beeram, D Yeritsyan… - Aging …, 2021 - Wiley Online Library
Hutchinson‐Gilford progeria syndrome (HGPS) is a rare accelerated aging disorder most
notably characterized by cardiovascular disease and premature death from myocardial …

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 …