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 …

[HTML][HTML] Ageing–oxidative stress, PTMs and disease

T Ebert, N Tran, L Schurgers, P Stenvinkel… - Molecular aspects of …, 2022 - Elsevier
Post-translational modifications (PTMs) have been proposed as a link between the oxidative
stress-inflammation-ageing trinity, thereby affecting several hallmarks of ageing …

[HTML][HTML] New hallmarks of ageing: a 2022 Copenhagen ageing meeting summary

T Schmauck-Medina, A Molière, S Lautrup… - Aging (Albany …, 2022 - ncbi.nlm.nih.gov
Genomic instability, telomere attrition, epigenetic alterations, mitochondrial dysfunction, loss
of proteostasis, deregulated nutrient-sensing, cellular senescence, stem cell exhaustion …

Post-translational modification of GPX4 is a promising target for treating ferroptosis-related diseases

C Cui, F Yang, Q Li - Frontiers in Molecular Biosciences, 2022 - frontiersin.org
Glutathione peroxidase 4 (GPX4) is one of the most important antioxidant enzymes. As the
key regulator of ferroptosis, GPX4 has attracted considerable attention in the fields of cancer …

Pharmacological targeting of RAS: recent success with direct inhibitors

JP O'Bryan - Pharmacological research, 2019 - Elsevier
RAS has long been viewed as undruggable due to its lack of deep pockets for binding of
small molecule inhibitors. However, recent successes in the development of direct RAS …

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 …

SARS-CoV-2 non-structural proteins and their roles in host immune evasion

ZY Low, NZ Zabidi, AJW Yip, A Puniyamurti, VTK Chow… - Viruses, 2022 - mdpi.com
Coronavirus disease 2019 (COVID-19) has caused an unprecedented global crisis and
continues to threaten public health. The etiological agent of this devastating pandemic …

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 …

Multifaceted mechanisms of vascular calcification in aging

LA Pescatore, LF Gamarra… - … , thrombosis, and vascular …, 2019 - Am Heart Assoc
Approximately 20% of the world's population will be around or above 65 years of age by the
next decade. Out of these, 40% are suspected to have cardiovascular diseases as a cause …

In vivo cyclic induction of the FOXM1 transcription factor delays natural and progeroid aging phenotypes and extends healthspan

R Ribeiro, JC Macedo, M Costa, V Ustiyan… - Nature Aging, 2022 - nature.com
The FOXM1 transcription factor exhibits pleiotropic C-terminal transcriptional and N-terminal
non-transcriptional functions in various biological processes critical for cellular homeostasis …