Toshifumi Yokota, University of Alberta

Research

Muscular Dystrophy Spinal Muscular Atrophy Duchenne Muscular Dystrophy Antisense Therapy Fibrodysplasia Ossificans Progressiva Antisense Oligonucleotide-Mediated Therapy Neuromuscular Diseases Exon Skipping

Latest papers

FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.
RNA biology · 2026 · senior author
Lipid nanoparticle delivery of antisense gapmers attenuates pathology in a mouse model of facioscapulohumeral muscular dystrophy.
Molecular therapy : the journal of the American Society of Gene Therapy · 2026 · senior author
Molecular Diagnosis to Individualized Therapies in Rare Genetic Diseases: New Approach Methodologies, RNA Therapeutics, and the Case for a Human-First Filter.
Genes · 2026 · senior author

All 174 publications

Latest funding

Development and Optimization of DG9-Peptide-Conjugated Morpholino Oligomers for the Systemic Treatment of Spinal Muscular Atrophy
CIHR · 2026 · Nominated PI
$918,000
Advancing RNA Splicing Therapies for Duchenne Muscular Dystrophy: Optimizing Multi-Exon Skipping Strategies for Broader Clinical Applicability
CIHR · 2025 · Nominated PI
$986,850
Commercializing antisense oligonucleotide therapy targeting DUX4 for facioscapulohumeral muscular dystrophy (FSHD)
CIHR · 2025 · Nominated PI
$1,147,500

All 17 grants and awards

From the 150 most recent of 174 publications.

01

FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.

Moriyama H, Moriyama S, Yokota T

RNA biology2026Senior author

DOI: 10.1080/15476286.2026.2689117 PMID: 42324661

02

Lipid nanoparticle delivery of antisense gapmers attenuates pathology in a mouse model of facioscapulohumeral muscular dystrophy.

Anwar S, Chan KYT, Zia A, Beck SL, Moriyama H, Lim KRQ, Shah MNA, Haque US, Maruyama R, Witzigmann D, Echigoya Y, Bosnakovski D, Kyba M, Cullis PR, Yokota T

Molecular therapy : the journal of the American Society of Gene Therapy2026Senior author

DOI: 10.1016/j.ymthe.2026.05.010 PMID: 42198843

03

Molecular Diagnosis to Individualized Therapies in Rare Genetic Diseases: New Approach Methodologies, RNA Therapeutics, and the Case for a Human-First Filter.

Anwar S, Yokota T

Genes2026Senior author

DOI: 10.3390/genes17070780 PMID: 42510820

04

DMD-Null mice exhibit severe muscle weakness, impaired regeneration, and deficient satellite cell function.

Wilton-Clark H, Shah MNA, Leckie J, Hernandez Rodriguez S, Al-Aghbari A, Zhabyeyev P, Maruyama R, Aoki Y, Oudit GY, Yokota T

Proceedings of the National Academy of Sciences of the United States of America2026Senior author

DOI: 10.1073/pnas.2606703123 PMID: 42301789

05

Single-base 2'OMe-modified LNA and MOE gapmers selectively silence ACVR1 R206H in fibrodysplasia ossificans progressiva.

Anwar S, Hay S, Moriyama H, Mir F, Maruyama R, Yokota T

Molecular therapy. Nucleic acids2026Senior author

DOI: 10.1016/j.omtn.2026.102937 PMID: 42327837

06

The Multi-System Roles of Dp71 Dystrophin Isoforms in Duchenne Muscular Dystrophy.

Wilton-Clark H, Raza A, Yokota T

Muscles (Basel, Switzerland)2026Senior author

DOI: 10.3390/muscles5020043 PMID: 42347484

07

Building CRISPR-Based Gene-Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics.

Rodriguez SH, Yokota T

Genes2026Senior author

DOI: 10.3390/genes17060631 PMID: 42353791

08

RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.

Kuroda T, Yokota T

Biomolecules2026Senior author

DOI: 10.3390/biom16060794 PMID: 42352260

09

Application of Omics Analysis in the Clinical Practice and Research of Transthyretin Amyloidosis.

Moriyama H, Shaikh FA, Yokota T

Genes2026Senior author

DOI: 10.3390/genes17030333 PMID: 41898867

10

From Genomic Diagnosis to Personalized RNA Medicine: Advances in Next-Generation Sequencing and N-of-1 Antisense Oligonucleotide Therapies for Rare Genetic Diseases.

Rodriguez Carstens P, Moriyama H, Yokota T

Genes2026Senior author

DOI: 10.3390/genes17030318 PMID: 41898852

Development and Optimization of DG9-Peptide-Conjugated Morpholino Oligomers for the Systemic Treatment of Spinal Muscular Atrophy

Nominated PICIHR2026Project Grant

Principal investigators: Yokota, Toshifumi

Keywords: Spinal Muscular Atrophy

$918,000

Advancing RNA Splicing Therapies for Duchenne Muscular Dystrophy: Optimizing Multi-Exon Skipping Strategies for Broader Clinical Applicability

Nominated PICIHR2025Project Grant

Principal investigators: Yokota, Toshifumi

Keywords: Duchenne Muscular Dystrophy

$986,850

Commercializing antisense oligonucleotide therapy targeting DUX4 for facioscapulohumeral muscular dystrophy (FSHD)

Nominated PICIHR2025Project Grant

Principal investigators: Yokota, Toshifumi

Keywords: Muscular Dystrophy

$1,147,500

Community-Guided Development of Genetic Therapies for Spinal Bulbar Muscular Atrophy: ASO Delivery Optimization and CRISPRa-Mediated Isoform Upregulation

SupervisorCIHR2025DRA : Canada Graduate Research Scholarship - Doctoral Program (CGRS D)

Principal investigators: Leckie, Jamie N

Keywords: Androgen Receptor; Antisense Oligonucleotide; Crispr Activation; Isoform Upregulation; Spinal Bulbar Muscular Atrophy

$120,000

Indigenous ways of knowing spinal bulbar muscular atrophy and directing translational research

Co-investigatorSSHRC2023New Frontiers in Research Fund - Exploration

Principal investigators: Pfeffer, Gerald

Keywords: spinal bulbar muscular atrophy; Kennedy disease; Indigenous health; qualitative methods; translational research; biomarkers

$250,000

Evaluating the Therapeutic Potential of Antisense Oligonucleotides and CRISPR Activation in Spinal Bulbar Muscular Atrophy, with a Focus on its Prevalence in Canadian Indigenous Populations

SupervisorCIHR2023CIHR Strategic Master's Award - First Nations, Inuit and/or Métis Health

Principal investigators: Leckie, Jamie N

Keywords: Androgen Receptor; Antisense Oligonucleotides; Ar Isoform 2; Crispr Activation; Spinal Bulbar Muscular Atrophy

$17,500

Development of allele-specific antisense oligonucleotide therapy for Fibrodysplasia ossificans progressiva

Nominated PICIHR2022Project Grant

Principal investigators: Yokota, Toshifumi

Keywords: Fibrodysplasia Ossificans Progressiva

$753,526

The Role of CSF2RB1 Ligands in Cancer-Associated Cachexia

Co-investigatorCIHR2022Project Grant

Principal investigators: Clemente Casares, Javier

Keywords: Cachexia; Cancer; Csf2rb1 Ligands; Inflammation; Mdsc

$918,000

Neutrophil Responses in Doxorubicin-Induced Myopathy

Co-investigatorCIHR2022Project Grant

Principal investigators: Clemente Casares, Javier

Keywords: Doxorubicin; Inflammation; Myopathy; Wasting

$879,750

Commercializing a novel platform for delivery of antisense oligonucleotide-mediated therapy

Nominated PICIHR2022Project Grant

Principal investigators: Yokota, Toshifumi

Keywords: Antisense Oligonucleotide-Mediated Therapy

$1,009,800

From CIHR, NSERC and SSHRC funding decisions: CIHR since 2008, NSERC since 1991 and SSHRC since 1998, including their latest published competition results.

Frequent collaborators

Medical Genetics
Oncology
Medical Microbiology and Immunology

Co-authors at University of Alberta, colored by department. Thicker lines mean more shared papers; select anyone to open their profile and their own map.

Sue Tsai

Medical Microbiology and Immunology

1 shared papers, latest 2023

Write to this professor

A short, specific email works best. This draft uses one of their recent papers; replace the parts in brackets with your own details before sending.

Applying for

Paper to mentionMention their CIHR grant from 2026

Subject: Prospective PhD student: Muscular Dystrophy Dear Professor Yokota, My name is [your name], and I am a [your current degree or role] at [your institution]. I am applying for a PhD position starting in [term and year]. I read your paper "FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook." (2026), and it connects closely to my own work on [your research topic]. [One or two sentences on what you did and what you learned.] I also saw your CIHR-funded project "Development and Optimization of DG9-Peptide-Conjugated Morpholino Oligomers for the Systemic Treatment of Spinal Muscular Atrophy" (2026), and I would be glad to contribute to work in that direction. Are you accepting new PhD students for [term and year]? I have attached my CV and transcripts, and I would be glad to talk at a time that suits you. Thank you for your time. Kind regards, [your name]

Toshifumi Yokota

Research

Latest papers

Latest funding

FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.

Lipid nanoparticle delivery of antisense gapmers attenuates pathology in a mouse model of facioscapulohumeral muscular dystrophy.

Molecular Diagnosis to Individualized Therapies in Rare Genetic Diseases: New Approach Methodologies, RNA Therapeutics, and the Case for a Human-First Filter.

DMD-Null mice exhibit severe muscle weakness, impaired regeneration, and deficient satellite cell function.

Single-base 2'OMe-modified LNA and MOE gapmers selectively silence ACVR1 R206H in fibrodysplasia ossificans progressiva.

The Multi-System Roles of Dp71 Dystrophin Isoforms in Duchenne Muscular Dystrophy.

Building CRISPR-Based Gene-Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics.

RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.

Application of Omics Analysis in the Clinical Practice and Research of Transthyretin Amyloidosis.

From Genomic Diagnosis to Personalized RNA Medicine: Advances in Next-Generation Sequencing and N-of-1 Antisense Oligonucleotide Therapies for Rare Genetic Diseases.

Indigenous ways of knowing spinal bulbar muscular atrophy and directing translational research

Frequent collaborators

Kristi Baker

Sue Tsai