Fragile X-associated neuropsychiatric disorders (FXAND)

RJ Hagerman, D Protic, A Rajaratnam… - Frontiers in …, 2018 - frontiersin.org
Fragile X syndrome (FXS) is caused by the full mutation (> 200 CGG repeats) in the Fragile
X Mental Retardation 1 (FMR1) gene. It is the most common inherited cause of intellectual …

Modeling Neurodegenerative Disorders in Drosophila melanogaster

H Bolus, K Crocker, G Boekhoff-Falk… - International Journal of …, 2020 - mdpi.com
Drosophila melanogaster provides a powerful genetic model system in which to investigate
the molecular mechanisms underlying neurodegenerative diseases. In this review, we …

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 …

CUG initiation and frameshifting enable production of dipeptide repeat proteins from ALS/FTD C9ORF72 transcripts

R Tabet, L Schaeffer, F Freyermuth, M Jambeau… - Nature …, 2018 - nature.com
Expansion of G4C2 repeats in the C9ORF72 gene is the most prevalent inherited form of
amyotrophic lateral sclerosis and frontotemporal dementia. Expanded transcripts undergo …

Non-Mendelian inheritance patterns and extreme deviation rates of CGG repeats in autism

DJ Annear, G Vandeweyer, A Sanchis-Juan… - Genome …, 2022 - genome.cshlp.org
As expansions of CGG short tandem repeats (STRs) are established as the genetic etiology
of many neurodevelopmental disorders, we aimed to elucidate the inheritance patterns and …

CGG repeat-associated non-AUG translation utilizes a cap-dependent scanning mechanism of initiation to produce toxic proteins

MG Kearse, KM Green, A Krans, CM Rodriguez… - Molecular cell, 2016 - cell.com
Repeat-associated non-AUG (RAN) translation produces toxic polypeptides from nucleotide
repeat expansions in the absence of an AUG start codon and contributes to …

CGG repeats trigger translational frameshifts that generate aggregation-prone chimeric proteins

SE Wright, CM Rodriguez, J Monroe, J **ng… - Nucleic acids …, 2022 - academic.oup.com
CGG repeat expansions in the FMR1 5'UTR cause the neurodegenerative disease Fragile X-
associated tremor/ataxia syndrome (FXTAS). These repeats form stable RNA secondary …

RNA-binding proteins in neurodegeneration: mechanisms in aggregate

EG Conlon, JL Manley - Genes & development, 2017 - genesdev.cshlp.org
Neurodegeneration is a leading cause of death in the developed world and a natural, albeit
unfortunate, consequence of longer-lived populations. Despite great demand for therapeutic …

Native functions of short tandem repeats

SE Wright, PK Todd - Elife, 2023 - elifesciences.org
Over a third of the human genome is comprised of repetitive sequences, including more than
a million short tandem repeats (STRs). While studies of the pathologic consequences of …

New pathologic mechanisms in nucleotide repeat expansion disorders

CM Rodriguez, PK Todd - Neurobiology of disease, 2019 - Elsevier
Tandem microsatellite repeats are common throughout the human genome and intrinsically
unstable, exhibiting expansions and contractions both somatically and across generations …