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 …
[HTML][HTML] Ageing–oxidative stress, PTMs and disease
Post-translational modifications (PTMs) have been proposed as a link between the oxidative
stress-inflammation-ageing trinity, thereby affecting several hallmarks of ageing …
stress-inflammation-ageing trinity, thereby affecting several hallmarks of ageing …
[HTML][HTML] New hallmarks of ageing: a 2022 Copenhagen ageing meeting summary
Genomic instability, telomere attrition, epigenetic alterations, mitochondrial dysfunction, loss
of proteostasis, deregulated nutrient-sensing, cellular senescence, stem cell exhaustion …
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 …
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 …
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 …
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
Coronavirus disease 2019 (COVID-19) has caused an unprecedented global crisis and
continues to threaten public health. The etiological agent of this devastating pandemic …
continues to threaten public health. The etiological agent of this devastating pandemic …
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 …
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 …
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
The FOXM1 transcription factor exhibits pleiotropic C-terminal transcriptional and N-terminal
non-transcriptional functions in various biological processes critical for cellular homeostasis …
non-transcriptional functions in various biological processes critical for cellular homeostasis …