Drug repurposing for rare diseases

HI Roessler, NVAM Knoers, MM van Haelst… - Trends in …, 2021 - cell.com
Currently, there are about 7000 identified rare diseases, together affecting 10% of the
population. However, fewer than 6% of all rare diseases have an approved treatment option …

Vascular stiffness in aging and disease

SF Vatner, J Zhang, C Vyzas, K Mishra… - Frontiers in …, 2021 - frontiersin.org
The goal of this review is to provide further understanding of increased vascular stiffness
with aging, and how it contributes to the adverse effects of major human diseases …

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 …

Lonafarnib: first approval

S Dhillon - Drugs, 2021 - Springer
Lonafarnib (Zokinvy™) is an orally active farnesyltransferase inhibitor developed by Eiger
BioPharmaceuticals under license from Merck & Co. for the treatment of hepatitis D virus …

A not-so-ancient grease history: click chemistry and protein lipid modifications

KF Suazo, KY Park, MD Distefano - Chemical reviews, 2021 - ACS Publications
Protein lipid modification involves the attachment of hydrophobic groups to proteins via
ester, thioester, amide, or thioether linkages. In this review, the specific click chemical …

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 …

Telomerase therapy reverses vascular senescence and extends lifespan in progeria mice

A Mojiri, BK Walther, C Jiang, G Matrone… - European Heart …, 2021 - academic.oup.com
Abstract Aims Hutchinson-Gilford progeria syndrome (HGPS) is an accelerated ageing
syndrome associated with premature vascular disease and death due to heart attack and …

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 …

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 …

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 …