Excited to share our new article Molecular Diagnosis to Individualized Therapies in Rare Genetic Diseases: New Approach Methodologies, RNA Therapeutics, and the Case for a Human-First Filter www.mdpi.com/3965648 #mdpigenes

Excited to share that our article, “Building CRISPR-Based Gene-Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics,” has been featured on the cover of the June 2026 issue of Genes. Congratulations to the team.https://www.mdpi.com/2073-4425/17/6

Congratulations to Harry and our team on our new PNAS publication. We report that DMD-Null mice exhibit severe muscle weakness, impaired regeneration, and deficient satellite cell function. grateful to @wchriuofa.bsky.social @heartandstroke.bsky.social www.pnas.org/doi/10.1073/...

Congratulations to Harry and Alishba on our new review: The Multi-System Roles of Dp71 Dystrophin Isoforms in Duchenne Muscular Dystrophy We summarize Dp71 roles in brain, retina, skeletal/cardiac muscle, and emerging therapeutic strategies www.mdpi.com/3930242 @mdpiopenaccess.bsky.social

Grateful to Folio for highlighting our work on RNA-based therapeutics for FOP. We sincerely appreciate the support from the Canadian FOP Network, iFOPA, @wchriuofa.bsky.social, Rare Disease Foundation, @stollerykids.bsky.social and all partners advancing FOP research. www.ualberta.ca/en/news/spot...

Congrats to Takayuki on receiving a CIHR Postdoc Award through the Canada Impact+ program, a wonderful recognition of Dr. Kuroda’s potential and an exciting opportunity to further strengthen international research collaboration @ualberta.bsky.social www.ualberta.ca/en/news/news...

Excited to share our new review article Building CRISPR-Based Gene-Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics We discuss how gene-editing technologies may help advance personalized therapeutic strategies www.mdpi.com/2073-4425/17...

Excited to share our new review in Biomolecules: “RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.” This article highlights recent progress in antisense oligonucleotides and gene-modifying approaches Read the article here: www.mdpi.com/2218-273X/16...

Excited to share our new paper on LNP delivery of antisense gapmers for FSHD. Repeated systemic LNP-gapmer treatment reduced DUX4, improved muscle function, and ameliorated pathology in an FSHD mouse model. Congrats to Saeed and all collaborators! @ualberta.bsky.social www.cell.com/molecular-th...

Proud to share that Alishba and Krish from the Yokota Lab have been awarded 2026 Alberta Innovates Summer Research Studentships. Their projects will advance our lab’s work in personalized RNA-targeted therapies for rare diseases. Congratulations! www.ualberta.ca/en/current-s...

Excited to share our new bioRxiv preprint on eSkip2, an AI-driven framework for exon-skipping ASO design. eSkip2 uses transfer learning from the genome foundation model HyenaDNA plus ASO and SNV splicing data to prioritize exon–intron target regions. www.biorxiv.org/content/10.6...

Congratulations to Umme Sabrina Haque, PhD candidate in the Yokota Lab, for winning the People’s Choice Award at the Western Canadian 3MT® Finals in Regina! We are very proud of Sabrina and her outstanding presentation, “A Cure with a Passport.” @ualberta.bsky.social www.youtube.com/watch?v=Px3Q...

www.preprints.org/manuscript/2... New preprint online: Building Gene Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics With Sebastian, we review CRISPR, base editing, prime editing, and scalable paths toward personalized therapies for rare genetic diseases.

www.preprints.org/manuscript/2... New preprint now online: RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies We review emerging antisense and gene-modifying approaches for skeletal muscle and neuromuscular diseases.

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/...