Antisense oligonucleotides: the next frontier for treatment of neurological disorders
C Rinaldi, MJA Wood - Nature Reviews Neurology, 2018 - nature.com
Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …
modulate protein expression over two decades ago; however, progress translating these …
Sequencing and characterizing short tandem repeats in the human genome
HA Tanudisastro, IW Deveson, H Dashnow… - Nature Reviews …, 2024 - nature.com
Short tandem repeats (STRs) are highly polymorphic sequences throughout the human
genome that are composed of repeated copies of a 1–6-bp motif. Over 1 million variable …
genome that are composed of repeated copies of a 1–6-bp motif. Over 1 million variable …
[HTML][HTML] Converging mechanisms in ALS and FTD: disrupted RNA and protein homeostasis
Breakthrough discoveries identifying common genetic causes for amyotrophic lateral
sclerosis (ALS) and frontotemporal dementia (FTD) have transformed our view of these …
sclerosis (ALS) and frontotemporal dementia (FTD) have transformed our view of these …
An atlas of alternative splicing profiles and functional associations reveals new regulatory programs and genes that simultaneously express multiple major isoforms
Alternative splicing (AS) generates remarkable regulatory and proteomic complexity in
metazoans. However, the functions of most AS events are not known, and programs of …
metazoans. However, the functions of most AS events are not known, and programs of …
[HTML][HTML] RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention
A hexanucleotide GGGGCC repeat expansion in the noncoding region of the C9ORF72
gene is the most common genetic abnormality in familial and sporadic amyotrophic lateral …
gene is the most common genetic abnormality in familial and sporadic amyotrophic lateral …
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
R Kole, AR Krainer, S Altman - Nature reviews Drug discovery, 2012 - nature.com
Here, we discuss three RNA-based therapeutic technologies exploiting various
oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have …
oligonucleotides that bind to RNA by base pairing in a sequence-specific manner yet have …
[HTML][HTML] Disruption of RNA metabolism in neurological diseases and emerging therapeutic interventions
RNA binding proteins are critical to the maintenance of the transcriptome via controlled
regulation of RNA processing and transport. Alterations of these proteins impact multiple …
regulation of RNA processing and transport. Alterations of these proteins impact multiple …
The myotonic dystrophies: molecular, clinical, and therapeutic challenges
B Udd, R Krahe - The Lancet Neurology, 2012 - thelancet.com
Myotonic dystrophy is the most common type of muscular dystrophy in adults and is
characterised by progressive myopathy, myotonia, and multiorgan involvement. Two …
characterised by progressive myopathy, myotonia, and multiorgan involvement. Two …
[HTML][HTML] Distal alternative last exons localize mRNAs to neural projections
Spatial restriction of mRNA to distinct subcellular locations enables local regulation and
synthesis of proteins. However, the organizing principles of mRNA localization remain …
synthesis of proteins. However, the organizing principles of mRNA localization remain …
[HTML][HTML] C9orf72 hexanucleotide repeat associated with amyotrophic lateral sclerosis and frontotemporal dementia forms RNA G-quadruplexes
Large expansions of a non-coding GGGGCC-repeat in the first intron of the C9orf72 gene
are a common cause of both amyotrophic lateral sclerosis (ALS) and frontotemporal …
are a common cause of both amyotrophic lateral sclerosis (ALS) and frontotemporal …