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 …
Therapeutic targeting of long non-coding RNAs in cancer
Long non-coding RNAs (lncRNAs) represent a significant population of the human
transcriptome. Many lncRNAs exhibit cell-and/or tissue/tumor-specific expression, making …
transcriptome. Many lncRNAs exhibit cell-and/or tissue/tumor-specific expression, making …
Non-AUG translation: a new start for protein synthesis in eukaryotes
Although it was long thought that eukaryotic translation almost always initiates at an AUG
start codon, recent advancements in ribosome footprint mapping have revealed that non …
start codon, recent advancements in ribosome footprint mapping have revealed that non …
Small molecule recognition of disease-relevant RNA structures
SM Meyer, CC Williams, Y Akahori, T Tanaka… - Chemical Society …, 2020 - pubs.rsc.org
Targeting RNAs with small molecules represents a new frontier in drug discovery and
development. The rich structural diversity of folded RNAs offers a nearly unlimited reservoir …
development. The rich structural diversity of folded RNAs offers a nearly unlimited reservoir …
Discovery of a biomarker and lead small molecules to target r (GGGGCC)-associated defects in c9FTD/ALS
A repeat expansion in C9ORF72 causes frontotemporal dementia and amyotrophic lateral
sclerosis (c9FTD/ALS). RNA of the expanded repeat (r (GGGGCC) exp) forms nuclear foci or …
sclerosis (c9FTD/ALS). RNA of the expanded repeat (r (GGGGCC) exp) forms nuclear foci or …
Fragile X-associated tremor/ataxia syndrome—features, mechanisms and management
RJ Hagerman, P Hagerman - Nature Reviews Neurology, 2016 - nature.com
Many physicians are unaware of the many phenotypes associated with the fragile X
premutation, an expansion in the 5′ untranslated region of the fragile X mental retardation …
premutation, an expansion in the 5′ untranslated region of the fragile X mental retardation …
A superfolding Spinach2 reveals the dynamic nature of trinucleotide repeat–containing RNA
RL Strack, MD Disney, SR Jaffrey - Nature methods, 2013 - nature.com
Imaging RNA in living cells is a challenging problem in cell biology. One strategy for
genetically encoding fluorescent RNAs is to express them as fusions with Spinach, an'RNA …
genetically encoding fluorescent RNAs is to express them as fusions with Spinach, an'RNA …
Sequence-based design of bioactive small molecules that target precursor microRNAs
Oligonucleotides are designed to target RNA using base pairing rules, but they can be
hampered by poor cellular delivery and nonspecific stimulation of the immune system. Small …
hampered by poor cellular delivery and nonspecific stimulation of the immune system. Small …
Advances in clinical and molecular understanding of the FMR1 premutation and fragile X-associated tremor/ataxia syndrome
R Hagerman, P Hagerman - The Lancet Neurology, 2013 - thelancet.com
Fragile X syndrome, the most common heritable form of cognitive impairment, is caused by
epigenetic silencing of the fragile X (FMR1) gene owing to large expansions (> 200 repeats) …
epigenetic silencing of the fragile X (FMR1) gene owing to large expansions (> 200 repeats) …
Inforna 2.0: a platform for the sequence-based design of small molecules targeting structured RNAs
MD Disney, AM Winkelsas, SP Velagapudi… - ACS chemical …, 2016 - ACS Publications
The development of small molecules that target RNA is challenging yet, if successful, could
advance the development of chemical probes to study RNA function or precision …
advance the development of chemical probes to study RNA function or precision …