Toshifumi Yokota

1. Shah MNA et al (2025) DG9-PMO Boosts Nuclear Localization and DMD Exon 44 Skipping, Enhancing Muscle Function and Cardioprotection. Nat Commun. 16(1):4477.(IF=14.7)
    
2. Irigoien E et al (2025) AOC 1044 Induces Exon 44 Skipping and Restores Dystrophin Protein in Preclinical Models of Duchenne Muscular Dystrophy. Nucleic Acids Res. 53(6):gkaf241. (IF=19.16)
   
   
3. Anwar S et al (2025) Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells. Mol Ther Nucleic Acids. 36(1):102443.(IF=10.20)
    
4. Aslesh et al (2023) DG9 peptide-conjugated morpholino rescues phenotype in SMA model mice by reaching the CNS through a single subcutaneous administration. JCI Insight. e160516. (IF=9.484)
    
5. Lim et al (2022) Development of DG9 peptide-conjugated single- and multi-exon skipping antisense oligonucleotides for the treatment of Duchenne muscular dystrophy. Proc. Natl. Acad. Sci. U.S.A.119 (9) e2112546119 (IF=12.78)
    
6. Chiba et al (2021) eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping. Nucleic Acids Res. 49(W1): W193-W198. (IF=19.16)
   
   
7. Lim et al (2021) DUX4 transcript knockdown with antisense 2’-O-methoxyethyl gapmers for the treatment of facioscapulohumeral muscular dystrophy. Mol Ther. 29: 848-58. (IF=12.90)
    
8. Lim et al (2020) Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy. Proc. Natl. Acad. Sci. U.S.A. 117: 16509-16515.
   
   
9. Echigoya et al (2019) Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene. Mol Ther. 27: 2005-2017.
    
10. Lim et al (2019) Efficacy of multi-exon skipping treatment in Duchenne muscular dystrophy dog model neonates. Mol Ther. 27: 76-86.
     
11. Lee et al (2018) Identification of novel antisense-mediated exon skipping targets in DYSF for therapeutic treatment of dysferlinopathy. Mol Ther Nucleic Acids.13: 596-604. (IF=10.20)
     
12. Echigoya et al (2017) Effects of systemic multi-exon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy. Proc. Natl. Acad. Sci. U.S.A. 114: 4213-8.
    
    
13. Echigoya et al (2017) Quantitative antisense screening and optimization for exon 51 skipping in Duchenne muscular dystrophy. Mol Ther. 25: 2561-2572.

The Yokota Lab develops next-generation RNA- and genome-targeted therapies for rare neuromuscular and genetic diseases. The research program integrates molecular biology, bioinformatics, and translational medicine to advance precision therapeutics for conditions such as Duchenne and Becker muscular dystrophy (DMD/BMD), dysferlinopathy, facioscapulohumeral muscular dystrophy (FSHD), spinal muscular atrophy (SMA), fibrodysplasia ossificans progressiva (FOP), spinal and bulbar muscular atrophy (SBMA), and other severe neuromuscular disorders.

The group’s multidisciplinary work includes the development of antisense oligonucleotide (ASO) drugs, peptide–PMO conjugates (e.g., DG9-PMO), CRISPR-based genome editing, and personalized “n-of-1” RNA therapeutics designed for individual patients. Machine learning models are also employed to optimize ASO sequence design and delivery, accelerating therapeutic discovery through computational prediction and validation.

In addition to therapeutic development, the lab explores the role of aquaporins in muscle physiology, particularly aquaporin-4 (AQP4), a water channel that is markedly reduced in dystrophic muscle. Ongoing research aims to define the molecular mechanisms governing AQP4 recruitment to the sarcolemma and its impact on muscle function, providing new insights into the pathophysiology of muscular dystrophy and related disorders.

By combining fundamental research with translational application, the Yokota Lab is committed to improving the lives of individuals affected by genetic disease through innovation, collaboration, and precision medicine.

The Yokota Lab welcomes postdoctoral, graduate, and undergraduate trainees pursuing RNA-based and gene-editing therapies for neuromuscular diseases. Training includes antisense design, CRISPR editing, Disease modelling, and AI-driven prediction of therapeutic efficacy. Collaborative projects with international and industry partners offer broad translational exposure.