Advances in oligonucleotide drug delivery

TC Roberts, R Langer, MJA Wood - Nature reviews Drug discovery, 2020 - nature.com
Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Physiological functions and pathobiology of TDP‐43 and FUS/TLS proteins

A Ratti, E Buratti - Journal of neurochemistry, 2016 - Wiley Online Library
The multiple roles played by RNA binding proteins in neurodegeneration have become
apparent following the discovery of TAR DNA binding protein 43 kDa (TDP‐43) and fused in …

Brain expression quantitative trait locus and network analyses reveal downstream effects and putative drivers for brain-related diseases

N de Klein, EA Tsai, M Vochteloo, D Baird, Y Huang… - Nature …, 2023 - nature.com
Identification of therapeutic targets from genome-wide association studies (GWAS) requires
insights into downstream functional consequences. We harmonized 8,613 RNA-sequencing …

Population-scale tissue transcriptomics maps long non-coding RNAs to complex disease

OM de Goede, DC Nachun, NM Ferraro… - Cell, 2021 - cell.com
Long non-coding RNA (lncRNA) genes have well-established and important impacts on
molecular and cellular functions. However, among the thousands of lncRNA genes, it is still …

Long non-coding RNA: its evolutionary relics and biological implications in mammals: a review

JK Dhanoa, RS Sethi, R Verma, JS Arora… - Journal of animal …, 2018 - Springer
The central dogma of gene expression propounds that DNA is transcribed to mRNA and
finally gets translated into protein. Only 2–3% of the genomic DNA is transcribed to protein …

The emerging field of epigenetics in neurodegeneration and neuroprotection

JY Hwang, KA Aromolaran, RS Zukin - Nature Reviews Neuroscience, 2017 - nature.com
Epigenetic mechanisms—including DNA methylation, histone post-translational
modifications and changes in nucleosome positioning—regulate gene expression, cellular …

MeCP2: the genetic driver of Rett syndrome epigenetics

KV Good, JB Vincent, J Ausió - Frontiers in Genetics, 2021 - frontiersin.org
Mutations in methyl CpG binding protein 2 (MeCP2) are the major cause of Rett syndrome
(RTT), a rare neurodevelopmental disorder with a notable period of developmental …

Long non-coding RNA (lncRNA) roles in cell biology, neurodevelopment and neurological disorders

V Aliperti, J Skonieczna, A Cerase - Non-coding RNA, 2021 - mdpi.com
Development is a complex process regulated both by genetic and epigenetic and
environmental clues. Recently, long non-coding RNAs (lncRNAs) have emerged as key …

Competing endogenous RNAs, non-coding RNAs and diseases: an intertwined story

U Ala - Cells, 2020 - mdpi.com
MicroRNAs (miRNAs), a class of small non-coding RNA molecules, are responsible for RNA
silencing and post-transcriptional regulation of gene expression. They can mediate a fine …

Defective control of pre–messenger RNA splicing in human disease

B Chabot, L Shkreta - Journal of Cell Biology, 2016 - rupress.org
Examples of associations between human disease and defects in pre–messenger RNA
splicing/alternative splicing are accumulating. Although many alterations are caused by …