RNA drugs and RNA targets for small molecules: principles, progress, and challenges
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …
On the wrong DNA track: Molecular mechanisms of repeat-mediated genome instability
Expansions of simple tandem repeats are responsible for almost 50 human diseases, the
majority of which are severe, degenerative, and not currently treatable or preventable. In this …
majority of which are severe, degenerative, and not currently treatable or preventable. In this …
CGG repeat RNA G-quadruplexes interact with FMRpolyG to cause neuronal dysfunction in fragile X-related tremor/ataxia syndrome
S Asamitsu, Y Yabuki, S Ikenoshita, K Kawakubo… - Science …, 2021 - science.org
Fragile X-related tremor/ataxia syndrome (FXTAS) is a neurodegenerative disease caused
by CGG triplet repeat expansions in FMR1, which elicit repeat-associated non-AUG (RAN) …
by CGG triplet repeat expansions in FMR1, which elicit repeat-associated non-AUG (RAN) …
The integrated stress response and phosphorylated eukaryotic initiation factor 2α in neurodegeneration
S Bond, C Lopez-Lloreda, PJ Gannon… - … of Neuropathology & …, 2020 - academic.oup.com
The proposed molecular mechanisms underlying neurodegenerative pathogenesis are
varied, precluding the development of effective therapies for these increasingly prevalent …
varied, precluding the development of effective therapies for these increasingly prevalent …
The clinical trial landscape in amyotrophic lateral sclerosis—Past, present, and future
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease marked by
progressive loss of muscle function. It is the most common adult‐onset form of motor neuron …
progressive loss of muscle function. It is the most common adult‐onset form of motor neuron …
Trinucleotide CGG repeat diseases: an expanding field of polyglycine proteins?
M Boivin, N Charlet-Berguerand - Frontiers in Genetics, 2022 - frontiersin.org
Microsatellites are repeated DNA sequences of 3–6 nucleotides highly variable in length
and sequence and that have important roles in genomes regulation and evolution. However …
and sequence and that have important roles in genomes regulation and evolution. However …
C9orf72 ALS/FTD dipeptide repeat protein levels are reduced by small molecules that inhibit PKA or enhance protein degradation
Abstract Intronic GGGGCC (G4C2) hexanucleotide repeat expansion within the human
C9orf72 gene represents the most common cause of familial forms of amyotrophic lateral …
C9orf72 gene represents the most common cause of familial forms of amyotrophic lateral …
A 5′ UTR GGN repeat controls localisation and translation of a potassium leak channel mRNA through G-quadruplex formation
Abstract RNA G-quadruplexes (G4s) are secondary structures proposed to function as
regulators of post-transcriptional mRNA localisation and translation. G4s within some …
regulators of post-transcriptional mRNA localisation and translation. G4s within some …
Drug screen in iPSC-Neurons identifies nucleoside analogs as inhibitors of (G4C2) n expression in C9orf72 ALS/FTD
Summary An intronic (G 4 C 2) n expansion in C9orf72 causes amyotrophic lateral sclerosis
and frontotemporal dementia primarily through gain-of-function mechanisms: the …
and frontotemporal dementia primarily through gain-of-function mechanisms: the …
Advancements in clinical RNA therapeutics: Present developments and prospective outlooks
PE Saw, E Song - Cell Reports Medicine, 2024 - cell.com
RNA molecules have emerged as promising clinical therapeutics due to their ability to
target" undruggable" proteins or molecules with high precision and minimal side effects …
target" undruggable" proteins or molecules with high precision and minimal side effects …