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Recent publications

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.

Klein AF. et al, (2019), J Clin Invest

Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7.

Curtis HJ. et al, (2017), Nucleic Acids Res, 45, 7870 - 7885

Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles.

O'Loughlin AJ. et al, (2017), Mol Ther, 25, 1580 - 1587

C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia.

Aoki Y. et al, (2017), Brain, 140, 887 - 897

Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.

Varela MA. et al, (2016), Eur J Hum Genet, 24, 271 - 276

Identification of sequences common to more than one therapeutic target to treat complex diseases: simulating the high variance in sequence interactivity evolved to modulate robust phenotypes.

Varela MA., (2015), BMC Genomics, 16

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.

Betts CA. et al, (2015), Sci Rep, 5

Corrigendum: Cross-talking noncoding RNAs contribute to cell-specific neurodegeneration in SCA7.

Tan JY. et al, (2015), Nat Struct Mol Biol, 22

Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.

Hammond SM. et al, (2014), Mol Ther Nucleic Acids, 3

Cross-talking noncoding RNAs contribute to cell-specific neurodegeneration in SCA7.

Tan JY. et al, (2014), Nat Struct Mol Biol, 21, 955 - 961

Epigenetics and ncRNAs in brain function and disease: mechanisms and prospects for therapy.

Varela MA. et al, (2013), Neurotherapeutics, 10, 621 - 631

Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective.

Fernandes SA. et al, (2013), J Nucleic Acids, 2013

Natural Antisense Makes Sense for Gene-specific Activation in Brain.

Varela MA. et al, (2012), Mol Ther Nucleic Acids, 1

Genetic heterogeneity in natural beds of the razor clam Ensis siliqua revealed by microsatellites

(2012), Journal of the Marine Biological Association of the United Kingdom, 92, 171 - 177

Heterogeneous distribution of SNPs in the human genome: microsatellites as predictors of nucleotide diversity and divergence.

Varela MA. and Amos W., (2010), Genomics, 95, 151 - 159

Temporal genetic variation of microsatellite markers in the razor clam ensis arcuatus (bivalvia: Pharidae)

(2009), Journal of the Marine Biological Association of the United Kingdom, 89, 1703 - 1707

Evidence for nonindependent evolution of adjacent microsatellites in the human genome.

Varela MA. and Amos W., (2009), J Mol Evol, 68, 160 - 170

Unstable repeat expansion and human disease

Varela MA., (2009), The Human Genome: Features, Variations and Genetic Disorders, 197 - 211

Heterogeneous nature and distribution of interruptions in dinucleotides may indicate the existence of biased substitutions underlying microsatellite evolution.

Varela MA. et al, (2008), J Mol Evol, 66, 575 - 580

Development of microsatellite markers in the razor clam Solen marginatus (Bivalvia: Solenidae)

(2007), Journal of the Marine Biological Association of the United Kingdom, 87, 977 - 978

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