Molecular mechanisms underlying nucleotide repeat expansion disorders

I Malik, CP Kelley, ET Wang, PK Todd - Nature reviews Molecular cell …, 2021 - nature.com
The human genome contains over one million short tandem repeats. Expansion of a subset
of these repeat tracts underlies over fifty human disorders, including common genetic …

Decoding ALS: from genes to mechanism

JP Taylor, RH Brown Jr, DW Cleveland - Nature, 2016 - nature.com
Amyotrophic lateral sclerosis (ALS) is a progressive and uniformly fatal neurodegenerative
disease. A plethora of genetic factors have been identified that drive the degeneration of …

RNA phase transitions in repeat expansion disorders

A Jain, RD Vale - Nature, 2017 - nature.com
Expansions of short nucleotide repeats produce several neurological and neuromuscular
disorders including Huntington disease, muscular dystrophy, and amyotrophic lateral …

[HTML][HTML] Rescue of fragile X syndrome neurons by DNA methylation editing of the FMR1 gene

XS Liu, H Wu, M Krzisch, X Wu, J Graef, J Muffat… - Cell, 2018 - cell.com
Fragile X syndrome (FXS), the most common genetic form of intellectual disability in males,
is caused by silencing of the FMR1 gene associated with hypermethylation of the CGG …

Non-AUG translation: a new start for protein synthesis in eukaryotes

MG Kearse, JE Wilusz - Genes & development, 2017 - genesdev.cshlp.org
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 …

The organizing principles of eukaryotic ribosome recruitment

J Pelletier, N Sonenberg - Annual review of biochemistry, 2019 - annualreviews.org
The stage at which ribosomes are recruited to messenger RNAs (mRNAs) is an elaborate
and highly regulated phase of protein synthesis. Upon completion of this step, a ribosome is …

RAN translation at C9orf72-associated repeat expansions is selectively enhanced by the integrated stress response

KM Green, MR Glineburg, MG Kearse… - Nature …, 2017 - nature.com
Repeat-associated non-AUG (RAN) translation allows for unconventional initiation at
disease-causing repeat expansions. As RAN translation contributes to pathogenesis in …

RNA G-quadruplexes: emerging mechanisms in disease

A Cammas, S Millevoi - Nucleic acids research, 2017 - academic.oup.com
Abstract RNA G-quadruplexes (G4s) are formed by G-rich RNA sequences in protein-coding
(mRNA) and non-coding (ncRNA) transcripts that fold into a four-stranded conformation …

C9ORF72 GGGGCC repeat-associated non-AUG translation is upregulated by stress through eIF2α phosphorylation

W Cheng, S Wang, AA Mestre, C Fu, A Makarem… - Nature …, 2018 - nature.com
Hexanucleotide repeat expansion in C9ORF72 is the most frequent cause of both
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Here we …

Non-AUG translation initiation in mammals

DE Andreev, G Loughran, AD Fedorova… - Genome Biology, 2022 - Springer
Recent proteogenomic studies revealed extensive translation outside of annotated protein
coding regions, such as non-coding RNAs and untranslated regions of mRNAs. This non …