Very pleased that our team has received a $918,000 CIHR Project Grant to develop DG9-PMO as a systemic, body-wide treatment for spinal muscular atrophy. Grateful to our trainees, collaborators, CIHR and the SMA community. #SMA #CIHR #RNAtherapeutics www.ualberta.ca/en/news/news...

U of A experts aim to advance life-changing health research

Health researchers at the University of Alberta will lead projects exploring next-generation treatments, advanced AI diagnostics, health-care equity and chronic disease recovery, thanks to $20.4 milli...

ualberta.ca

Our 2026 Friends of Garrett Cumming Research Chair Report is now available. Highlights include advances in DG9-mediated oligonucleotide delivery, Indigenous-partnered SBMA research, personalized N-of-1 therapies, and more than $2.5M in new CIHR funding. sites.google.com/ualberta.ca/...

Yokota Lab - Research Chair

Advancing the Future of Muscular Dystrophy Research A Legacy Inspired by Garrett Cumming Garrett Cumming was three years old when he was diagnosed with Duchenne muscular dystrophy (DMD), a severe gene...

sites.google.com

Great news! Our review on patient-customized N-of-1 antisense oligonucleotide therapy has been selected as a Genes Editorial Choice. It has reached >12,000 readers and >20 citations. Congratulations to Harry Wilton-Clark and Eric Yan! doi.org/10.3390/gene... #Nof1 #RareDisease

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...

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

Anwar and colleagues report that lipid nanoparticle (LNP) delivery significantly enhances the efficacy and safety of antisense gapmers targeting DUX4 in facioscapulohumeral muscular dystrophy. These p...

cell.com

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...

Darke Hall - 3MT

YouTube video by Darke Hall Society

youtube.com

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...

Single-base 2'OMe-modified LNA and MOE gapmers selectively silence ACVR1R206H in FOP, improving muscle and tendon bioavailability and safety

Anwar and colleagues report a chemically optimized antisense gapmer strategy that selectively silences the pathogenic ACVR1R206H allele in fibrodysplasia ossificans progressiva, sparing the wild-type ...

cell.com

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...

WCHRI 2026 Summer Studentship results_20mar2026

Results of the 2026 Summer Studentship Competition WCHRI Summer Studentship program aims to encourage a new generation of talented, engaged young researchers to pursue careers in women’s and/or chil...

docs.google.com

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/

【Press Release】日本希少疾患コンソーシアム2025年度年会 湘南アイパークにて開催～ 「N-of‑1+」アプローチで切り拓く希少疾患の個別化医療 ～ | ニュース | 湘南ヘルスイノベーションパーク (湘南アイパーク)

Press ReleasePDF版はこちら2026年1月29日報道関係者各位アイパークインスティチュート株式会社日本希少疾患コンソーシアム2025年度年会 湘南アイパークにて開催～ 「N-of‑1+」...

shonan-ipark.com

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

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