Lightnews — Scholar-powered news

Toshi Yokota @tyokota.bsky.social · 1d

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

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Toshi Yokota @tyokota.bsky.social · 1d

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

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Toshi Yokota @tyokota.bsky.social · 8d

📢 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

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Toshi Yokota @tyokota.bsky.social · 11d

🎉 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

Toshi Yokota @tyokota.bsky.social · 12d

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

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Toshi Yokota @tyokota.bsky.social · 22d

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

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Toshi Yokota @tyokota.bsky.social · Sep 5

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

Toshi Yokota @tyokota.bsky.social · Sep 4

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

Toshi Yokota @tyokota.bsky.social · Aug 15

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

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Toshi Yokota @tyokota.bsky.social · Aug 7

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

Toshi Yokota @tyokota.bsky.social · Aug 6

🎉 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

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Toshi Yokota @tyokota.bsky.social · Aug 6

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

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Toshi Yokota @tyokota.bsky.social · Aug 5

🎉 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

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Toshi Yokota @tyokota.bsky.social · Jul 1

🚨 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

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Toshi Yokota @tyokota.bsky.social · May 27

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

Toshi Yokota @tyokota.bsky.social · May 18

🎉 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

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Toshi Yokota @tyokota.bsky.social · May 17

🚨 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

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Toshi Yokota @tyokota.bsky.social · May 15

🚨 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

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Toshi Yokota @tyokota.bsky.social · Apr 24

🚨 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

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Toshi Yokota @tyokota.bsky.social · Apr 21

Honored to receive a 2025 grant from Muscular Dystrophy Canada to advance lipid nanoparticle–based therapies for Friedreich ataxia and SBMA, with a focus on supporting Indigenous communities affected by neuromuscular disorders.
#FA #SBMA #GeneTherapy #IndigenousHealth muscle.ca/researchers/...

Translational Science and Clinical Research Grants – Muscular Dystrophy Canada

muscle.ca

Toshi Yokota @tyokota.bsky.social · Apr 19

Proud to have hosted Fulbright Scholar Omar Sheikh in the Yokota Lab! As a scientist living with Becker MD, his work on genetic therapies and advocacy for inclusion in science made a lasting impact. Read about his journey. #Fulbright #MuscularDystrophy #InclusionInSTEM miusa.org/resource/per...

From Lab to Life: Omar Sheikh's Canadian Fulbright Adventure - MIUSA

Omar Shake learned about life and his disability on a Fulbright research grant in Canada.

miusa.org

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Reposted by Toshi Yokota

Rika Maruyama @rikamaruyama.bsky.social · Apr 10

Our collaboration with Avidity Biosciences is officially published! Big congrats and thanks to everyone who made this possible!
academic.oup.com/nar/article/...

AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy

Abstract. Duchenne muscular dystrophy (DMD) is a severe disorder caused by mutations in the dystrophin gene, resulting in loss of functional dystrophin pro

academic.oup.com

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Toshi Yokota @tyokota.bsky.social · Mar 11

🚀 Exciting progress in gene therapy! Our team @ualberta.bsky.social has developed a molecule that restores the muscle-repairing protein dysferlin, offering hope for individuals with dysferlinopathy.
Grateful to our collaborators, patients, and supporters!
www.ualberta.ca/en/folio/202...

Gene therapy shows promise for treating rare muscle disease

A U of A research team has taken the first step toward proving its innovative gene therapy can successfully treat a rare genetic condition called dysferlinopathy that impairs the body’s ability to rep...

www.ualberta.ca

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Toshi Yokota @tyokota.bsky.social · Mar 4

🚀 Exciting news! Our team has been awarded a $1.3M CIHR grant to study Spinal-Bulbar Muscular Atrophy (SBMA) in Indigenous communities of the Prairie Provinces. Honored to collaborate with an incredible team of researchers & community leaders! @nmd4c.ca neuromuscularnetwork.ca/news/cihr-fu...

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Toshi Yokota @tyokota.bsky.social · Jan 1

✨ Exciting News! ✨
I'm thrilled to share that our article, "Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?", has been published in Expert Opinion on Drug Safety! 🚀🔬

Access the full article here: www.tandfonline.com/eprint/BFZQK...

Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?

Duchenne muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle weakness and eventual death due to cardiomyopathy or respiratory complications. Currently, there ...

www.tandfonline.com

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