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 …
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 …
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
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 …
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 …
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
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 …
ester, thioester, amide, or thioether linkages. In this review, the specific click chemical …
A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome
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 …
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
Abstract Aims Hutchinson-Gilford progeria syndrome (HGPS) is an accelerated ageing
syndrome associated with premature vascular disease and death due to heart attack and …
syndrome associated with premature vascular disease and death due to heart attack and …
Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome
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 …
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 …
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 …
by premature aging and death mainly because of myocardial infarction, stroke, or heart …