Tandem repeats mediating genetic plasticity in health and disease

AJ Hannan - Nature Reviews Genetics, 2018 - nature.com
Accumulating evidence suggests that many classes of DNA repeats exhibit attributes that
distinguish them from other genetic variants, including the fact that they are more liable to …

The expanding biology of the C9orf72 nucleotide repeat expansion in neurodegenerative disease

AR Haeusler, CJ Donnelly, JD Rothstein - Nature Reviews …, 2016 - nature.com
A nucleotide repeat expansion (NRE) within the chromosome 9 open reading frame 72
(C9orf72) gene was the first of this type of mutation to be linked to multiple neurological …

C9orf72 BAC mouse model with motor deficits and neurodegenerative features of ALS/FTD

Y Liu, A Pattamatta, T Zu, T Reid, O Bardhi… - Neuron, 2016 - cell.com
To define how the C9orf72 GGGGCC expansion mutation causes ALS/FTD and to facilitate
therapy development, a mouse model that recapitulates the molecular and phenotypic …

[HTML][HTML] Elimination of toxic microsatellite repeat expansion RNA by RNA-targeting Cas9

R Batra, DA Nelles, E Pirie, SM Blue, RJ Marina… - Cell, 2017 - cell.com
Microsatellite repeat expansions in DNA produce pathogenic RNA species that cause
dominantly inherited diseases such as myotonic dystrophy type 1 and 2 (DM1/2) …

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

C Lagier-Tourenne, M Baughn, F Rigo… - Proceedings of the …, 2013 - National Acad Sciences
Expanded hexanucleotide repeats in the chromosome 9 open reading frame 72 (C9orf72)
gene are the most common genetic cause of ALS and frontotemporal degeneration (FTD) …

Non-ATG–initiated translation directed by microsatellite expansions

T Zu, B Gibbens, NS Doty… - Proceedings of the …, 2011 - National Acad Sciences
Trinucleotide expansions cause disease by both protein-and RNA-mediated mechanisms.
Unexpectedly, we discovered that CAG expansion constructs express homopolymeric …

R loops and links to human disease

P Richard, JL Manley - Journal of molecular biology, 2017 - Elsevier
Aberrant R-loop structures are increasingly being realized as an important contributor to
human disease. R loops, which are mainly co-transcriptional, abundant RNA/DNA hybrids …

Disease-associated repeat instability and mismatch repair

MHM Schmidt, CE Pearson - DNA repair, 2016 - Elsevier
Expanded tandem repeat sequences in DNA are associated with at least 40 human genetic
neurological, neurodegenerative, and neuromuscular diseases. Repeat expansion can …

The disease-associated r (GGGGCC) n repeat from the C9orf72 gene forms tract length-dependent uni-and multimolecular RNA G-quadruplex structures

K Reddy, B Zamiri, SYR Stanley, RB Macgregor… - Journal of Biological …, 2013 - ASBMB
Certain DNA and RNA sequences can form G-quadruplexes, which can affect promoter
activity, genetic instability, RNA splicing, translation, and neurite mRNA localization …

Regulation of chromatin structure by long noncoding RNAs: focus on natural antisense transcripts

M Magistri, MA Faghihi, G St Laurent, C Wahlestedt - Trends in genetics, 2012 - cell.com
In the decade following the publication of the Human Genome, noncoding RNAs (ncRNAs)
have reshaped our understanding of the broad landscape of genome regulation. During this …