Miguel A. Varela

South Parks Road
Oxford, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 26 publications

Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.

Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.

Journal: Molecular therapy. Nucleic acids
Published: September 26, 2025

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1.

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1.

Journal: American journal of human genetics
Published: July 04, 2025

Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.

Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.

Journal: Brain : a journal of neurology
Published: August 10, 2024

TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition.

TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition.

Journal: Acta neuropathologica
Published: January 20, 2024

A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles.

A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles.

Journal: Nanoscale advances
Published: November 24, 2022

Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.

Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: November 18, 2022

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1.

Journal: International journal of molecular sciences
Published: September 19, 2022

Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.

Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.

Journal: Molecular therapy. Nucleic acids
Published: July 12, 2022

Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model.

Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model.

Journal: Molecular therapy. Nucleic acids
Published: September 15, 2021

Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.

Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.

Journal: Molecular therapy. Nucleic acids
Published: October 18, 2020

Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion.

Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: September 01, 2020

Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.

Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: September 01, 2020
Showing 1-12 of 26

Last Updated: 09/26/2026