Toshi Yokota
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tyokota.bsky.social
Toshi Yokota
@tyokota.bsky.social
Molecular Geneticist| RNA Therapeutics Innovator| FCAHS Canadian Academy Fellow| Distinguished Professor@UAlberta| Driving innovation in genetic medicine
Honoured that our FSHD antisense gapmer technology was recognized with a 2025 Innovation Award from
@ualberta.bsky.social
Technology Transfer Services, following its license to Facio BioTherapies and Children’s National Research Institute.
Grateful to our team and partners. #FSHD #RareDisease
November 23, 2025 at 4:06 AM
Excited to share our new preprint on NGS and N-of-1 antisense oligonucleotide therapies for rare genetic diseases.

We discuss how genomic diagnosis can directly inform personalized RNA medicines and N-of-1 ASO design. www.preprints.org/manuscript/2...
www.preprints.org
November 17, 2025 at 5:28 PM
Thrilled to share that Harry, MD/PhD student in our lab, has won 3rd Place at the Falling Walls Lab 2025 in Berlin for his project “Breaking the Wall of Rare Disease Therapies.” A remarkable achievement representing Canada and the @ualberta.bsky.social on a global stage!
falling-walls.com/lab
Falling Walls Lab
Throughout the year, 100 Falling Walls Labs take place all over the world. The winners pitch their breakthrough ideas at the Science Summit.
falling-walls.com
November 7, 2025 at 7:45 PM
Our Friends of Garrett Cumming Research Chair report 2025 is now live!
Learn more about our mission and ongoing research at UAlberta:
🔗 sites.google.com/ualberta.ca/...
Grateful to Muscular Dystrophy Canada and the Cumming family for their partnership.
Yokota Lab - Research Chair
Pioneering the Future of Muscular Dystrophy Research at the University of Alberta Garrett Cumming was only three years old when he was diagnosed with Duchenne Muscular Dystrophy (DMD), the most common...
sites.google.com
November 6, 2025 at 7:48 PM
Congratulations to Harry from our lab for receiving the Oral Presentation Award at the 2025 WCHRI Research Day!
His talk, “Customized exon skipping therapy to treat a young boy with DMD” highlights the promise of precision antisense therapy for rare diseases.
@wchriuofa.bsky.social
October 23, 2025 at 9:45 PM
Honoured to be named a University of Alberta Distinguished Professor.
Grateful to my students, collaborators, and colleagues worldwide for their dedication to advancing genetic medicine for rare diseases.
Proud to be part of this inspiring community. www.ualberta.ca/en/the-quad/...
Celebrating the Recipients of the 2025 Faculty Excellence Awards
www.ualberta.ca
October 15, 2025 at 2:07 PM
Exciting news!
Our latest Methods in Molecular Biology chapter is out:
“DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice.” Congratulations to Sabrina and Melissa for leading this important work.
link.springer.com/protocol/10....
DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease characterized by decreased expression of the survival of motor neuron 1 (SMN1) gene. Antisense oligonucleotide (ASO)-based therapies have been developed recently as a means of increasing the...
link.springer.com
October 12, 2025 at 8:48 AM
Thrilled to share our new Methods in Molecular Biology chapter. link.springer.com/protocol/10.... We describe in vivo methods for systemic DG9-PMO delivery, exon 51 skipping, and efficacy assessment in hDMDdel52;mdx mice. #DMD #RNAtherapeutics #ASO #DG9 #ExonSkipping @SpringerNature
DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene that leads to the loss of the dystrophin protein. Exon-skipping therapy with phosphorodiamidate morpholino oligomers (PMOs) has been shown to restore the disrupted...
link.springer.com
October 11, 2025 at 4:04 PM
📢 New publication from our lab!
“Local Non-Coding Regulatory Elements in Muscular Dystrophies” now out in IJMS.
Exploring how lncRNAs, miRNAs, and epigenetic regulators contribute to muscular dystrophies.
👉 www.mdpi.com/1422-0067/26... Congrats to Harry Wilton-Clark and Sebastian Rodriguez!
www.mdpi.com
October 4, 2025 at 4:21 PM
🎉 Exciting news! Our preprint “Local Non-Coding Regulatory Elements in the Muscular Dystrophies” (with Harry Wilton-Clark & Sebastian Hernandez Rodriguez) has already reached 100 downloads on @preprints.bsky.social Grateful for the growing interest. www.preprints.org/manuscript/2...
www.preprints.org
October 2, 2025 at 3:00 AM
Thrilled to share our new review article “Upgrading nucleic acid and antisense therapeutics: challenges, solutions, and future directions” published in Bioanalysis (Taylor & Francis). 🚀
50 free e-prints available here: www.tandfonline.com/doi/full/10....
Upgrading nucleic acid and antisense therapeutics: challenges, solutions, and future directions
Only a small fraction of disease-modifying proteins present druggable pockets for conventional small-molecule or biologic therapies, underscoring the urgent need for innovative strategies such as n...
www.tandfonline.com
September 30, 2025 at 3:51 PM
Excited to share that our new preprint is now online! 🚀
"Artificial Intelligence-Driven Design of Antisense Oligonucleotides for Precision Medicine in Neuromuscular Disorders" Grateful to my co-authors for their contributions.
👉 www.preprints.org/manuscript/2... www.preprints.org/manuscript/2...
www.preprints.org
September 21, 2025 at 2:04 AM
Thrilled to share that our MD/PhD student Harry Wilton-Clark won First Place at Falling Walls Lab Edmonton 2025 🏆👏
His talk, Breaking the Wall of Rare Disease Therapies, will now represent Edmonton on the world stage in Berlin! 🌍 #FallingWallsLab #RareDisease www.ualberta.ca/en/graduate-...
Falling Walls Lab Edmonton
www.ualberta.ca
September 5, 2025 at 2:08 AM
Excited to share our new preprint: Local Non-Coding Regulatory Elements in the Muscular Dystrophies 🚀
We review how #lncRNAs, #miRNAs, and epigenetic regulators shape disease mechanisms in #DMD, #FSHD, #LGMD & more. www.preprints.org/manuscript/2... #preprints via @Preprints_org
www.preprints.org
September 4, 2025 at 1:20 AM
New hope for Duchenne muscular dystrophy💙
Our team identified DG9, a peptide that boosts delivery of exon-skipping therapy to the heart — a key unmet need in DMD. This approach restored cardiac & skeletal muscle function in mice. www.ualberta.ca/en/medicine/... @wchriuofa.bsky.social
Researchers build on a breakthrough in treating muscular dystrophy
www.ualberta.ca
August 15, 2025 at 1:51 AM
pleased to share that our article "Report on the Rare Disease Consortium Japan Inaugural Symposium" is now published in the Journal of Neuromuscular Diseases.
doi.org/10.1177/2214...
This work highlights the dedication of RDCJ and partners to advancing research and collaboration in rare diseases.
Report on the rare disease consortium Japan inaugural symposium - July 18, 2023, shonan health innovation park, Japan - Toshifumi Yokota, Naoto Inukai, Hiroyuki Shibasaki, Harumasa Nakamura, Shinnichi...
The Rare Disease Consortium Japan (RDCJ) is a newly formalized cross-sector initiative launched to address the urgent and growing needs of individuals living wi...
doi.org
August 7, 2025 at 10:47 PM
🎉 Congratulations to our graduate student Umme Sabrina Haque on receiving the prestigious WCHRI Graduate Studentship!
Her work in medical genetics is helping advance therapies for neuromuscular disorders that affect children.
@wchriuofa.bsky.social www.wchri.org/our-impact/s...
Psychology graduate student explores link between social media and youth mental health through WCHRI support – WCHRI
From understanding self-perceptions to advancing disease treatments, WCHRI's graduate students are shaping the future of women's and children's health research.
www.wchri.org
August 6, 2025 at 2:55 PM
Excited to share our latest preprint:
"Cardiac Cell and Animal Models for Duchenne Muscular Dystrophy in the Era of Gene Therapy and Precision Medicine"
Now online at @PreprintsOrg!
🧬 www.preprints.org/manuscript/2...
#DMD #GeneTherapy #PrecisionMedicine #Cardiology #Preprint #MuscularDystrophy
www.preprints.org
August 6, 2025 at 1:25 AM
🎉 New Release! The 2nd Edition of Exon Skipping and Inclusion Therapies is now published with Springer Nature!
This edition highlights new breakthroughs in RNA-targeted therapies.
Grab your copy
link.springer.com/book/10.1007...
#ExonSkipping #RNAtherapeutics #GeneTherapy #DMD #SMA #SpringerNature
Exon Skipping and Inclusion Therapies
This book presents a collection of protocols reflecting the latest advancements in exon skipping and inclusion strategies.
link.springer.com
August 5, 2025 at 12:49 AM
🚨 New publication alert!

Our latest review on Brogidirsen & exon 44 skipping therapy for Duchenne muscular dystrophy is out in Genes. We discuss advances, challenges, and future directions for RNA-based therapies.

🔗 Read the full open-access article: www.mdpi.com/2073-4425/16...
www.mdpi.com
July 1, 2025 at 3:15 AM
www.preprints.org/manuscript/2... Excited to share our latest preprint:
🧬 “Brogidirsen and Exon 44 Skipping for DMD: Advances and Challenges in RNA-Based Therapy”
📄 www.preprints.org/manuscript/2...
Proud of Annie Tang’s excellent work leading this review. #DMD #RNAtherapy #exonskipping #ASO
www.preprints.org
May 27, 2025 at 12:58 AM
🎉 Proud to see our postdoc Dr. Hidenori Moriyama featured by @wchriuofa.bsky.social ! His work on improving ASO delivery for muscular dystrophy is bringing new hope to families. Thank you @stollerykids.bsky.social for supporting this research.
#RareDisease #WCHRI www.wchri.org/our-impact/p...
wchri.org
May 18, 2025 at 2:27 AM
🚨 Just featured in The Gateway!
Our lab's work on exon skipping & antisense therapy is helping pave the way for genetic treatments that target DMD and beyond. 📰 Read the article: thegatewayonline.ca/2025/05/u-of...
U of A researchers make advancements in gene therapy - The Gateway
U of A professor of medical genetics Toshifumi Yokota and the Yokota Lab are making advancements in gene therapy.
thegatewayonline.ca
May 17, 2025 at 8:15 PM
🚨 New publication in Nature Communications!
DG9–PMO improves nuclear delivery and exon skipping, restoring muscle & heart function in DMD models. A major step toward cardioprotective therapies.
doi.org/10.1038/s414...
Grateful to
@wchriuofa.bsky.social
@ualberta.bsky.social
DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function - Nature Communications
The authors show that the cell-penetrating peptide DG9 enhances PMO delivery to skeletal and cardiac muscles via multiple endocytic pathways. DG9-PMO significantly boosts dystrophin restoration and ca...
doi.org
May 15, 2025 at 1:03 AM
🚨 We’re hiring!
The Yokota Lab @ualberta.bsky.social is looking for a Postdoctoral Fellow to join our team developing cutting-edge genetic therapies for muscular dystrophy & rare diseases.
🌍 Edmonton, AB
🧪 3-year term (annual renewal)
Apply by July 31 👉 jrecin.jst.go.jp/seek/SeekJor...
JREC-IN | Postdoctoral Fellow (Yokota Lab)
イノベーション創出を担う研究人材のためのキャリア支援ポータルサイト
jrecin.jst.go.jp
April 24, 2025 at 2:40 AM