Congratulations to Sabrina from the Yokota Lab on receiving an Oral Presentation Award at NMHI Research Day 2026. Sabrina continues to do outstanding work, and I am very pleased to see her research recognized. Well deserved. @ualberta.bsky.social www.ualberta.ca/en/neuroscie...

Our new paper now online! Single-base 2'OMe-modified LNA and MOE gapmers selectively silence ACVR1R206H in #FOP, improving muscle and tendon bioavailability and safety Grateful to all collaborators, trainees and supporters @ualberta.bsky.social @wchriuofa.bsky.social www.cell.com/molecular-th...

We are pleased to share a new preprint from the Yokota Lab: The Multi-System Roles of Dp71 Dystrophin Isoforms in Duchenne Muscular Dystrophy This review discusses the importance of Dp71 dystrophin isoforms in the multi-system biology of DMD.　 www.preprints.org/manuscript/2...

I am delighted to share that Sabrina Haque from our lab has been named the 2026 3 Minute Thesis Champion at the University of Alberta for her talk, “A Cure with a Passport.” We are very proud of her. Congratulations, Sabrina! @ualberta.bsky.social www.ualberta.ca/en/graduate-...

Thrilled that Rida has secured a @wchriuofa.bsky.social Summer Studentship! She’ll be working with us on developing safer, whole-body ASO treatments for Spinal Muscular Atrophy. Thanks to @stollerykids.bsky.social for the support! docs.google.com/document/d/1...

Congrats to Hidenori and Faiyza on the publication of our review article in Genes Application of Omics Analysis in the Clinical Practice and Research of Transthyretin Amyloidosis This paper highlights how omics approaches are advancing both research and clinical practice www.mdpi.com/2073-4425/17...

Our review article is now published in Genes: From Genomic Diagnosis to Personalized RNA Medicine: Advances in Next-Generation Sequencing and N-of-1 Antisense Oligonucleotide Therapies for Rare Genetic Diseases www.mdpi.com/2073-4425/17... Congratulations to all co-authors.

日本希少疾患コンソーシアム（RDCJ）2025年度年会にて、「北米におけるN-of-1創薬の現状：患者個別ASO開発の実例と、規制・製造・実装に向けた課題」というタイトルで講演します! 北米での患者個別ASO（N-of-1）開発の実例を共有し、日本での制度設計・実装の議論に少しでも貢献できればと思います。 www.shonan-ipark.com/news/2551/

New preprint out! 📄 We examine the role of omics analysis in clinical practice and research for Transthyretin Amyloidosis. Check out the full work here: www.preprints.org/manuscript/2... #RareDisease #ATTR #Genomics #Bioinformatics @ualberta.bsky.social

Just published! 🎉 Our new review “Artificial Intelligence-Driven Design of Antisense Oligonucleotides for Precision Medicine in Neuromuscular Disorders” is now out. We discuss how computational pipelines can accelerate ASO design and support N-of-1 therapies. 🔗 Article: www.mdpi.com/2073-4425/16...

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

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...

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

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.

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

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/...

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....

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

📢 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!

🎉 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...

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....

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...

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-...

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

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

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.

🎉 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...

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

🎉 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

🚨 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.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

🎉 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...

🚨 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...

🚨 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

🚨 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...

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/...

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...

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

🚀 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...

🚀 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...

✨ 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...