Publications

a) Journal Articles (Peer-reviewed)

—2024—

117. Wilton-Clark H*, Yan E*, Yokota T.

Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases.

Genes. 2024, 15(7), 821

116. Haque US*,Kohut M*, Yokota T.

Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO

Curr Res Toxicol. 2024, 7: 100182

115. Leckie J*, Yokota T.

Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA.

Molecules. 2024, 29(11), 2658

114. Anwar S*,  Yokota T.

The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments.

Biomolecules. 2024,14(3):256

113. Tang A*, Yokota T. 

Duchenne muscular dystrophy: promising early-stage clinical trials to watch.

Expert Opin Investig Drugs. 2024, 33(3): 201-217

—2023—

112. Anwar S*,  Yokota T.

Navigating the Complex Landscape of Fibrodysplasia Ossificans Progressiva: From Current Paradigms to Therapeutic Frontiers.

Genes. 2023, 14(12): 2162

111.Wilton-Clark H*, Yokota T.

Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment.

Med. 2023, 4(12): 855-856

110.Haque US*, Yokota T.

Enhancing Antisense Oligonucleotide-Based Therapeutic Delivery with DG9, a Versatile Cell-Penetrating Peptide.

Cells. 2023, 12(19), 2395

109.Wilton-Clark H*, Yokota T.

CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy

Mol Ther Methods Clin Dev. 2023, 30, 500-501.

108.Wilton-Clark H*, Al-aghbari A*, Jessica Y*, Yokota T.

Advancing Epidemiology and Genetic Approaches for the Treatment of Spinal and Bulbar Muscular Atrophy: Focus on Prevalence in the  Indigenous Population of Western Canada.

Genes. 2023, 14(8), 1634.

107.Maruyama R*, Fiorillo A, Heier C, Duan D, Yokota T.

Editorial: Genome and transcriptome editing to understand and treat neuromuscular diseases.

Front Genome Ed. 2023, 14;5:1176699.

106.Shah MNA*, Yokota T.

Cardiac therapies for Duchenne muscular dystrophy.

Ther Adv Neurol Disord. 2023, 3;16:17562864231182934.

105.Dzierlega K*, Chakraborty M, Lee M, Soliman AM, Parker D, Khan S, Chan YT, Akbari M, Yokota T, Winer S, Baker K, Tsai S, Winer DA, Clemente-Casares X.

Activin A-Expressing Polymorphonuclear Myeloid-Derived Suppressor Cells Infiltrate Skeletal and Cardiac Muscle and Promote Cancer Cachexia

J Immunol. 2023,1;211(3):497-507.

104.Zhu A*, Chiba S, Shimizu Y, Kunitake A, Okuno Y, Aoki Y, Yokota T.

Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping

Pharmaceutics.  2023, 15(7), 1808.

103.Nakevska Z*, Yokota T.

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review.

Eur J Cell Biol.2023,102(2):151326.

102. Anwar S*, Mir F*, Yokota T.

Enhancing the effectiveness of oligonucleotide therapeutics using cell-penetrating peptide conjugation, chemical modification and carrier-based delivery strategies. 

Pharmaceutics. 2023, 15(4), 1130.

101. Aslesh T*, Erkut E*, Ren J, Lim KRQ*, Woo S*, Hatlevig S, Moulton HM, Gosgnach S, Greer J , Maruyama R*, Yokota T.

DG9 peptide-conjugated morpholino rescues phenotype in SMA model mice by reaching the CNS through a single subcutaneous administration. 

JCI Insight. 2023.e160516.

100. Wilton-Clark H*, Yokota T.

Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy

Pharmaceutics. 15(3), 778

99. Shirakaki S*, Roshmi RR*, Yokota T. 

Genetic approaches for the treatment of giant axonal neuropathy. 

J Pers Med. 2023, 13(1) 91. 

98. Aslesh T*, Al-aghbari A*, Yokota T. 

Assessing the role of Aquaporin 4 in skeletal muscle function. 

Int J Mol Sci. 2023, 24(2) 1489

—2022— 

97. Wilton-Clark H*, Yokota T. 

Biological and genetic therapies for the treatment of Duchenne muscular dystrophy. 

Expert Opin Biol Ther. 2022, 23, 49-59. 

96. Sheikh O*, Yokota T. 

Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update. 

Arch Toxicol. 2022; 96(1):1-9.

95. Yin H, Lin C, Han G, Jia L, Zhao Y, Song J, Ran N, Yokota T, Seow Y. 

Cardio-respiratory and phenotypic rescue of dystrophin/utrophin-deficient mice by combination therapy.

EMBO Reports. 2022, 23(6):e53955. 10.15252/embr.202153955

94. Maruyama R*, Nguyen Q*, Roshmi RR*, Touznik A*, Yokota T.

Allele-Selective Locked Nucleic Acid Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation. 

Nucleic Acid Ther. 2022; 32(3):185-193. 10.1089/nat.2021.0009

93. Erkut E*, Yokota T. 

CRISPR Therapeutics for Duchenne Muscular Dystrophy.

Int J Mol Sci. 2022, 23(3):1832. 10.3390/ijms23031832

92. Lim KRQ*, Woo S*, Melo D*, Huang Y*, Dzierlega K*, Shah A*, Aslesh T*, Roshmi RR*, Echigoya Y*, Maruyama R*, Moulton HM, Yokota T.

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. 2022; 119(9): e2112546119. 10.1073/pnas.2112546119

91. Wilton-Clark H*, Yokota T. 

Antisense and gene therapy options for Duchenne muscular dystrophy arising from mutations in the N-terminal hotspot. 

Genes. 2022; 13(2): 257. 10.3390/genes13020257

90. Aslesh T*, Yokota T.

Restoring SMN expression: An overview of the therapeutic developments for the treatment of spinal muscular atrophy. 

Cells. 2022; 11(3): 417. 10.3390/cells11030417

—2021— 

89. Sheikh O*, Yokota T.

Developing DMD therapeutics: a review of the effectiveness of corticosteroids, stop-codon readthrough, and exon-skipping therapies

Expert Opin Investig Drugs. 2021, 30(2):167-176. 10.1080/13543784.2021.1868434

88. Wilton-Clark H*, Yokota T.

Casimersen for Duchenne muscular dystrophy

Drugs Today (Barc). 2021, 57(12):707-717. 10.1358/dot.2021.57.12.3352740

87. Echigoya Y*, Trieu N*, Duddy W, Moulton HM, Yin H, Partridge TA, Hoffman EP, Kornegay JN, Rohret FA, Rogers CS, Yokota T.

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping.

Int J Mol Sci. 2021, 22(23):13065. 10.3390/ijms222313065

86. Lim KRQ*, Shah MNA*, Woo S*, Wilton-Clark H*, Zhabyeyev P, Wang F, Maruyama R*, Oudit GY, Yokota T.

Natural History of a Mouse Model Overexpressing the Dp71 Dystrophin Isoform

Int J Mol Sci. 2021, 22(23):12617. 10.3390/ijms222312617

85. Roshmi RR*, Yokota T.

Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience

Clin Pharmacol. 2021, 13:235-242. 10.2147/CPAA.S288842

84. Sheikh O*, Yokota T.

Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping, Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy

BioDrugs. 2021, 35(4):389-399. 10.1007/s40259-021-00486-7

83. Aslesh T*, Erkut E*,  Yokota T. 

Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing. 

Expert Opin Biol Ther. 2021, 21(8):1049-1061. 10.1080/14712598.2021.1872539

82. Chiba S*, Lim K*, Sheri N*, Anwar S*, Erkut E*, Shah A*, Aslesh T*, Woo S*, Sheikh O*, Maruyama R*, Takano H, Kunitake K, Duddy W, Okuno Y, Aoki Y, Yokota T. 

eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping

Nucleic Acids Res. 2021, 49(W1):W193-W198. 10.1093/nar/gkab442

81. Lim K*, Yokota T. 

Genetic approaches for the treatment of facioscapulohumeral muscular dystrophy.

Front Pharmacol. 2021, 12:642858. 10.3389/fphar.2021.642858

80. Tone Y, Mamchaoui K, Tsoumpra MK, Hashimoto Y, Terada R, Maruyama R*, Gait MJ, Arzumanov AA, McClorey G, Imamura M, Takeda S, Yokota T, Wood MJA, Mouly V, Aoki Y.

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides.

Nucleic Acid Ther. 2021, 31(2):172-181. 10.1089/nat.2020.0907

79. Lim K*, Bittel A, Maruyama R*, Echigoya Y*, Nguyen Q*,  Huang Y*, Dzierlega K*, Zhang A, Chen Y, Yokota T. 

DUX4 transcript knockdown with antisense 2’-O-methoxyethyl gapmers for the treatment of facioscapulohumeral muscular dystrophy.

Mol Ther. 2021, 29(2): 848-58. 10.1016/j.ymthe.2020.10.010

78. Anwar  S*, He M*, Lim K*, Maruyama R*, Yokota T.

A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis.

J Pers Med. 2021, 11(1):46. 10.3390/jpm11010046

—2020— 

77. Lim KRQ*, Nguyen Q*, Yokota T. 

DUX4 signalling in the pathogenesis of facioscapulohumeral muscular dystrophy. 

Int J Mol Sci. 2020, 21:729 10.3390/ijms21030729

76. Lim K*, Nguyen Q*, Yokota T.

Genotype–Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry

J Pers Med. 2020, 10 (4), 241 10.3390/jpm10040241

75. Lim K*, Maruyama R*, Echigoya Y*, Nguyen Q*, Khawaja H, Chandra S, Jones T, Jones P, Chen Y, Yokota T. 

Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy. 

Proc Natl Acad Sci U S A. 2020, 117 (28), 16509-16515. 10.1073/pnas.1909649117

*In this study, we demonstrated for the first time that antisense-mediated gene knockdown against the DUX4 gene improved the muscle structure and function in cell and mouse models of facioscapulohumeral muscular dystrophy (FSHD), the third most common form of muscular dystrophy.

74. van Westering TLE, Johansson HJ, Hanson B, Coenen-Stass AML, Lomonosova Y, Tanihata J, Motohashi N, Yokota T, Takeda S, Lehtiö J, Wood MJA, El Andaloussi S, Aoki Y, Roberts TC. 

Mutation-independent proteomic signatures of pathological progression in murine models of Duchenne muscular dystrophy. 

Mol Cell Proteomics. 2020, 19(12):2047-2067. 10.1074/mcp.RA120.002345

73. Dzierlega K*, Yokota T. 

Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. 

Gene Ther. 2020, 27(9):407-416. 10.1038/s41434-020-0156-6

72. Maruyama R*, Yokota T. 

Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases. 

J Pers Med. 2020, 10(3): E129. 10.3390/jpm10030129

71. Sheikh O*, Yokota T. 

Advances in Genetic Characterization and Genotype–Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized Medicine Era. 

J Pers Med. 2020, 10(3): 111. 10.3390/jpm10030111

70. Bittel AJ, Sreetama SC, Bittel DC, Horn A, Novak JS, Yokota T, Zhang A, Maruyama R*, Lim KRQ*, Jaiswal JK, Chen Y.

Membrane Repair Deficit in Facioscapulohumeral Muscular Dystrophy. 

Int J Mol Sci. 2020, 21(15), 5575. 10.3390/ijms21155575

*We designed and provided antisense oligonucleotides for this study, which promoted membrane repair in dystrophic muscle cells against laser injury.

69. Lim KRQ*, Sheri N*, Nguyen Q*, Yokota T. 

Cardiac Involvement in Dystrophin-Deficient Females: Current Understanding and Implications for the Treatment of Dystrophinopathies. 

Genes (Basel). 2020, 11(7): 765. 10.3390/genes11070765

68. Anwar S*, Yokota T. 

Golodirsen for Duchenne muscular dystrophy. 

Drugs of Today. 2020, 56 (8), 491-504. 10.1358/dot.2020.56.8.3159186

67. Lim KRQ*, Nguyen Q*, Dzierlega K*, Huang Y*, Yokota T. 

CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy. 

Genes (Basel). 2020, 11(3): 342. 10.3390/genes11030342

66. Nguyen Q*, Lim KRQ*, Yokota T. 

Genome editing for the understanding and treatment of inherited cardiomyopathies. 

Int J Mol Sci. 2020, 21:733 10.3390/ijms21030733

—2019— 

65. Shimizu-Motohashi Y, Komaki H, Motohashi N, Takeda S, Yokota T, Aoki Y. 

Restoring dystrophin expression in Duchenne muscular dystrophy: Current status of therapeutic approaches. 

J Pers Med. 2019, 9(1):1. 10.3390/jpm9010001

64. Sato M, Shiba N, Miyazaki D, Shiba Y, Echigoya Y*, Yokota T, Takizawa H, Aoki Y Takeda S, Nakamura A.

 Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes. 

Biochem Biophys Res Commun. 2019, 520 (1), 179-185. 10.1016/j.bbrc.2019.09.095

63. Roshmi R*, Yokota T. 

Viltolarsen (NS-65/NCNP-01) for the treatment of Duchenne muscular dystrophy. 

Drugs of Today. 2019, 55 (10), 627-639. 10.1358/dot.2019.55.10.3045038

62. Echigoya Y*, Lim K*, Nagata T, Kuraoka M, Kobayashi M, Aoki Y, Partridge T, Maruyama R*, Takeda S, Yokota T. 

Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene. 

Mol Ther. 2019, 27(11): 2005-17. 10.1016/j.ymthe.2019.07.012

*For the first time, we demonstrated rescue of dystrophin expression using a cocktail of antisense oligonucleotides which is applicable to 47% of Duchenne muscular dystrophy patients.

61. Hwang J*, Yokota T. 

Recent advancements in exon skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies. 

Expert Rev Mol Med. 2019, 21: e5 10.1017/erm.2019.5

60. Nguyen Q*, Lim K*, Yokota T. 

Current Understanding and Treatment of Cardiac and Skeletal Muscle Pathology in Laminin-α2 Chain-Deficient Congenital Muscular Dystrophy. 

Appl Clin Genet. 2019, 12:113-130.  10.2147/TACG.S187481

59. Nguyen Q*, Yokota T. (2019) Antisense Oligonucleotides for the Treatment of Cardiomyopathy in Duchenne Muscular Dystrophy. Am J Transl Res. 11(3):1202-1218.

58. Lim K*, Echigoya Y*, Nagata T, Kuraoka M, Kobayashi M, Aoki Y, Partridge T, Maruyama R*, Takeda S, Yokota T. 

Efficacy of multi-exon skipping treatment in Duchenne muscular dystrophy dog model neonates. 

Mol Ther. 2019, 27(1): 76-86. 10.1016/j.ymthe.2018.10.011

*For the first time, we demonstrated rescue of muscle function using cocktail antisense oligonucleotides in a large animal model of Duchenne muscular dystrophy.

57. Miyatake S, Mizobe Y, Tsoumpra M, Lim KRQ*, Hara Y, Shabanpoor F, Yokota T, Takeda S, Aoki Y. 

Scavenger receptor class A1 mediates uptake of morpholino antisense oligonucleotide into dystrophic skeletal muscle. 

Mol Ther Nucleic Acids. 2019, 14:520-535. 10.1016/j.omtn.2019.01.008

—2018— 

56. Aslesh T*, Maruyama R*, Yokota T. 

Skipping multiple exons to treat DMD - promises and challenges. 

Biomedicines. 2018, 6(1), 1 10.3390/biomedicines6010001

55. Echigoya Y*, Lim K*, Nakamura A, Yokota T. 

Multiple exon skipping in the DMD hot spots: Prospects and challenges. 

J Pers Med. 2018, 8(4):41.  10.3390/jpm8040041

54. Lim KRQ*, Yoon C*, Yokota T. 

Applications of CRISPR/Cas9 Exon Skipping for Duchenne Muscular Dystrophy.

J Pers Med. 2018, 8(4):38. 10.3390/jpm8040038

53. Gordish-Dressman H, Willmann R, Dalle Pazze L, Kreibich A, van Putten M, Heydemann A, Bogdanik L, Lutz L, Davies K, Demonbruen AR, Duan D, Elsey D, Fukada S, Girgenrath M, Patrick Gonzalez J, Grounds MD, Nichols A, Partridge T, Passini M, Sanarica F, Schnell FJ, Wells DJ, Yokota T, Young CS, Zhong Z, Spurney C, Spencer M, De Luca A, Nagaraju K, Aartsma-Rus A. 

A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic- First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd mdx /J (D2/mdx) model in the context of the classic C57BL/10ScSn-Dmd mdx /J (Bl10/mdx). 

J Neuromuscul Dis. 2018, 5:407-417. 10.3233/JND-180324

52. Lee JJA*, Maruyama R, Duddy W, Sakurai H, Yokota T. 

Identification of novel antisense-mediated exon skipping targets in DYSF for therapeutic treatment of dysferlinopathy. 

Mol Ther Nucleic Acids. 2018, 13:596-604. 10.1016/j.omtn.2018.10.004

51. Lee JJA*, Echigoya Y*, Saito T, Duddy W, Aoki Y, Takeda S, Yokota T. 

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts. 

PLoS ONE. 2018, 13(5): e0197084. 10.1371/journal.pone.0197084

50. Maruyama R*, Touznik A*, Yokota T. 

Evaluation of exon inclusion induced by splice switching antisense oligonucleotides in SMA patient fibroblasts. 

J Vis Exp. 2018, 135. e57530. 10.3791/57530

49. Shimo T*, Hosoki K*, Nakatsuji Y, Yokota T, Obika S. 

A Novel Human Muscle Cell Model of Duchenne Muscular Dystrophy Created by CRISPR/Cas9 and Evaluation of Antisense-Mediated Exon Skipping. 

J Hum Genet. 2018, 63:89-92. 10.1038/s10038-017-0400-0 

48. Lee JJA*, Maruyama R*, Sakurai H, Yokota T. 

Cell membrane repair assay using a two-photon laser microscope. 

J Vis Exp. 2018, 131, e56999 10.3791/56999

—2017— 

47. Touznik A*, Maruyama R*, Echigoya Y*, Yokota T. 

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts. 

Sci Rep. 2017, 7(1):3672. 10.1038/s41598-017-03850-2

46. Echigoya Y*, Nakamura A, Aoki Y, Nagata T, Kuraoka M, Urasawa N, Panesar D*, Iversen P, Kole R, Maruyama R*, Partridge T, Takeda S, Yokota T. 

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 USA. 2017, 114 (16), 4213-4218. 10.1073/pnas.1613203114

* We designed novel antisense oligonucleotides conjugated with cell-penetrating peptides and demonstrated rescue of cardiac function accompanied by dystrophin expression in a dog model of Duchenne muscular dystrophy.

45. Echigoya Y*, Lim K*, Trieu N*, Bao B*, Miskew B*, Vila MC, Novak JS, Hara Y, Lee J*, Touznik A*, Mamchaoui K, Aoki Y, Takeda S, Nagaraju K, Mouly V, Maruyama R*, Duddy W, Yokota T. 

Quantitative antisense screening and optimization for exon 51 skipping in Duchenne muscular dystrophy. 

Mol Ther. 2017, 25(11): 2561-2572. 10.1016/j.ymthe.2017.07.014

*We designed an in silico tool to design effective antisense oligonucleotides for exon skipping, and demonstrated that novel oligonucleotides designed by this tool are 10 times more effective compared to the FDA-approved oligo called Eteplirsen for the treatment of Duchenne muscular dystrophy.

44. Lim K*, Maruyama R*, Yokota T. 

Eteplirsen in the treatment of Duchenne muscular dystrophy. 

Drug Des Devel Ther. 2017, 11: 533–545. 10.2147/DDDT.S97635

*Top 0.1% citation according to Scopus

43. Nakamura A, Shiba N, Miyazaki D, Nishizawa H, Inaba Y, Fueki N, Maruyama R*, Echigoya Y*, Yokota T. 

Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy. 

J Hum Genet. 2017, 62, 459–463. 10.1038/jhg.2016.152

42. Nguyen Q*, Yokota T. 

Immortalized muscle cell model to test the exon skipping efficacy for Duchenne muscular dystrophy. 

J Pers Med. 2017, 7(4):13. 10.3390/jpm7040013

—2016— 

41. Rodrigues M*, Echigoya Y*, Maruyama R*, Lim K*, Fukada S, Yokota T. 

Impaired regenerative capacity and lower revertant fibre expansion in dystrophin deficient mdx muscles on DBA/2 background. 

Sci Rep. 2016, 6:38371. 10.1038/srep38371

40. Bao B*, Maruyama R*, Yokota T. 

Targeting RNA for the treatment of facioscapulohumeral muscular dystrophy. 

Intractable Rare Dis Res. 2016, 5(3):168-76. 10.5582/irdr.2016.01056

39. Nichols B*, Aoki Y, Kuraoka M, Lee JJA*, Takeda S, Yokota T. 

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy. 

J Vis Exp. 2016, 111: e53776 10.3791/53776

38. Nakamura A, Fueki N, Shiba N, Motoki H, Miyazaki D, Nishizawa H, Echigoya Y*, Yokota T, Aoki Y, Takeda S. 

Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy. 

J Hum Genet. 2016, 61(7):663-7. 10.1038/jhg.2016.28

37. Rodrigues M*, Echigoya Y*, Fukada S, Yokota T. 

Current Translational Research and Murine Models for Duchenne Muscular Dystrophy. 

J Neuromuscul Dis. 2016, 3(1): 29-48. 10.3233/JND-150113

36. Kamaludin A*, Smolarchuk C, Bischof JM, Eggert R, Greer JJ, Ren J Lee JJA*, Yokota T, Berry FB, Wevrick R. 

Muscle dysfunction caused by loss of Magel2 in a mouse model of Prader-Willi and Schaaf-Yang syndromes. 

Hum Mol Genet. 2016, 25(17):3798-3809. 10.1093/hmg/ddw225

—2015— 

35. Campbell C, Mabaya G, Johnston M, Gagnon C, Chapman K, Genge A, Jasmin B, Kothary R, Mah JK, Mcmillan HJ, Warman J, Yokota T, Korngut L (2015) 

Canadian Neuromuscular Diseases Network (can-nmd) A Collaborative Approach for Establishing World Class Outcomes for Neuromuscular Diseases in Canada. 

Muscle Nerve. 52: S11-12

34. Guncay A*, Yokota T. 

Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold? 

Future Med Chem. 2015, 7(13):1631-5. 10.4155/fmc.15.116

33. Pandey SN, Kesari A, Yokota T, Pandey GS. 

Muscular Dystrophy: Disease Mechanisms and Therapies. 

Biomed Res Int. 2015:456348 10.1155/2015/456348

32. Yu X*, Bao B*, Echigoya Y*, Yokota T. 

Dystrophin-deficient large animal models: translational research and exon skipping. 

Am J Transl Res. 2015, 7(8):1214-31.

31. Nichols B*, Takeda S, Yokota T. 

Nonmechanical roles of dystrophin and associated proteins in exercise, neuromuscular junctions, and brains. 

Brain Sci. 2015, 5: 275-298.  10.3390/brainsci5030275

30. Echigoya Y*, Mouly V, Garcia L, Yokota T (corresponding), Duddy W. 

In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy. 

PLoS ONE 2015, 10(3): e0120058 10.1371/journal.pone.0120058

29. Echigoya Y*, Aoki Y, Miskew B*, Panesar D*, Touznik A*, Nagata T, Tanihata J, Nakamura A, Nagaraju K, Yokota T. 

Long-term efficacy of systemic multi-exon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice. 

Mol Ther Nucleic Acids. 2015, 4: e225. 10.1038/mtna.2014.76

*We designed cocktail antisense oligonucleotides applicable to 45% of Duchenne muscular dystrophy patients and demonstrated long-term functional rescue in a mouse model.

28. Bao B*, Yokota T. 

Potential of antisense therapy for facioscapulohumeral muscular dystrophy. 

Expert Opin Orphan Drugs. 2015, 3(12):1365-1374. https://doi.org/10.1517/21678707.2015.1092868

—2014— 

27. Echigoya Y*, Yokota T. 

Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides. 

Nucleic Acid Ther., 2014, 24(1):57-68. 10.1089/nat.2013.0451

26. Pandey SN, Lee YC, Yokota T, Chen YW. 

Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice. 

Mol Ther. 2014, 22(2):390-6. 10.1038/mt.2013.263

25. Yokota T, Miyagoe-Suzuki Y, Ikemoto T, Takeda S. 

Alpha1-Syntrophin deficient mice exhibit impaired muscle force recovery after osmotic shock. 

Muscle Nerve, 2014, 49(5):728-35. 10.1002/mus.23990

24. Touznik A*, Lee J*, Yokota T. 

New developments in exon skipping and splice modulation therapy for neuromuscular diseases. 

Expert Opin Biol Ther. 2014, 14(6):809-19. 10.1517/14712598.2014.896335

—2013— 

23. Aoki Y, Nagata T, Yokota T, Nakamura A, Wood MJ, Partridge T, Takeda S. 

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin α2 chain-null congenital muscular dystrophy mice. 

Hum Mol Genet., 2013, 22(24):4914-28. 10.1093/hmg/ddt341

22. Lee J*, Yokota T. 

Antisense therapy in neurology. 

J Pers Med. 2013, 3, 144-176. 10.3390/jpm3030144

21. Echigoya Y*1, Lee J*1, Rodrigues M*1 (1equally contributed), Nagata T, Tanihata J, Nozohourmehrabad A*, Panesar D*, Miskew B*, Aoki Y, Yokota T. 

Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice. 

PLoS One. 2013, 8(7): e69194. 10.1371/journal.pone.0069194

20. Aoki Y, Yokota T, Wood MJ. 

Development of multiexon-skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy.

Biomed Res Int. 2013, 402369. 10.1155/2013/402369

—2012— 

19. Aoki Y, Yokota T (corresponding), Nagata T, Nakamura A, Tanihata J, Saito T, Duguez SMR, Nagaraju K, Hoffman EP, Partridge T, Takeda S. 

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery. 

Proc Natl Acad Sci USA. 2012, 109(34):13763-8. 10.1073/pnas.1204638109

*We designed cocktail antisense oligonucleotides applicable to 45% of Duchenne muscular dystrophy patients and demonstrated functional rescue in a mouse model for the first time.

18. Yokota T (corresponding), Duddy W, Echigoya Y*, Kolski H. 

Exon skipping for nonsense mutations in Duchenne muscular dystrophy: Too many mutations, too few patients? 

Expert Opin Biol Ther. 2012, 12(9):1141-52. 10.1517/14712598.2012.693469

17. Yokota T (corresponding), Nakamura A, Nagata T, Saito T, Kobayashi M, Aoki Y, Echigoya Y*, Partridge T, Hoffman E, Takeda S. 

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. 

Nucleic Acid Ther. 2012, 22(5):306-15. 10.1089/nat.2012.0368 *Featured Article

—2011— 

16. Hoffman EP, Bronson A, Baudy AR, Yokota T, Takeda S, Connor EM. 

Restoring dystrophin expression in Duchenne muscular dystrophy muscle: Progress in exon-skipping and stop codon read-through. 

Am J Pathol. 2011, 179(1):12-22. 10.1016/j.ajpath.2011.03.050

15. Taniguchi M, Kobayashi M, Kanagawa M, Yu C, Mori K, Oda T, Kuga A, Kurahashi H, Akman HO, Di Mauro S, Kaji R, Yokota T, Takeda S, Toda T. 

Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy. 

Nature. 2011, 478:127–131. 10.1038/nature10456 * Faculty of 1000 recommended paper (Factor 6) selection

*We designed cocktail antisense oligonucleotides to correct the effects of a mutated gene called fukutin and demonstrated systemic effects in a mouse model of congenital muscular dystrophy for the first time.

14. Lu QL, Yokota T, Takeda S, Garcia L, Muntoni F, Partridge T. 

The status of exon skipping as a therapeutic approach to Duchenne muscular dystrophy, 

Mol Ther. 2011, 19:9-15. 10.1038/mt.2010.219

—2010— 

13. Aoki Y, Nakamura A, Yokota T, Saito T, Okazawa H, Nagata T, Takeda S. 

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. 

Mol Ther. 2010, 18:1995-2005. 10.1038/mt.2010.186

12. Saito T, Nakamura A, Aoki Y, Yokota T, Okada T, Osawa M, Takeda S. 

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient. 

PLoS One. 2010; 5: e12239. 10.1371/journal.pone.0012239

—2009— 

11. Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S, Hoffman E. 

Efficacy of morpholino systemic exon-skipping in Duchenne dystrophy dogs. 

Ann. Neurol. 2009, 65:667-76. 10.1002/ana.21627 * Faculty of 1000 exceptional paper (Factor 10) selection 

*For the first time, we demonstrated rescue of dystrophin expression using cocktail antisense oligonucleotides in a large animal model of Duchenne muscular dystrophy.

10. Yokota T, Takeda S, Lu QL, Partridge T, Nakamura A, Hoffman E. 

A renaissance for antisense oligonucleotide drugs in neurology: Exon-skipping breaks new ground. 

JAMA Neurol (Formerly Arch. Neurol.), 2009, 66:32-8. 10.1001/archneurol.2008.540 *Cover image selection

—2008— 

9. Sato K, Yokota T, Ichioka S, Shibata M, Takeda S. 

Vasodilation of intramuscular arterioles under shear stress in dystrophin-deficient skeletal muscle is impaired through decreased nNOS expression. 

Acta Myol. 2008, 27:30-6.

—2007— 

8. Yokota T (corresponding), Duddy W, Partridge T. 

Optimizing exon skipping therapies for DMD, 

Acta Myol. 2007, 26:179-84.

7. Yokota T (corresponding), Emidio P, Duddy W, Kanneboyina N. 

Potential of exon skipping therapy for Duchenne muscular dystrophy, 

Expert Opin Biol Ther. 2007, 7:831-42. 10.1517/14712598.7.6.831

—2006— 

6. Yokota T, Lu QL, Morgan JE, Davies KE, Fisher R, Takeda S, Partridge T. 

Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as index of muscle regeneration. 

J Cell Sci. 2006, 119: 2679-87. 10.1242/jcs.03000

—2005— 

5. Lu QL, Rabinowitz A, Chen YC, Yokota T, Yin H, Alter J, Jadoon A, Bou-Gharios G, Partridge T. 

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. 

Proc Natl Acad Sci USA. 2005, 102:198-203. 10.1073/pnas.0406700102 *Cover image selection

* We designed antisense oligonucleotides targeting exon 23 in the Dmd gene and demonstrated systemic rescue of dystrophin in a mouse model for the first time.

—2004— 

4. Munehira Y, Ohnishi T, Kawamoto S, Furuya A, Shitara K, Imamura M, Yokota T, Takeda S, Amachi T, Matsuo M, Kioka N, Ueda K. 

Alpha1-syntrophin modulates turnover of ABCA1. 

J Biol Chem. 2004, 9; 279:15091-5. 10.1074/jbc.M313436200

—2002— 

3. Yokota T1, Hosaka Y1 (1Equally Contributed), Miyagoe-Suzuki Y, Yuasa K, Imamura M, Matsuda R, Ikemoto T, Kameya S, Takeda S. 

Alpha1-syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration, 

J Cell Biol. 2002, 158: 1097-1107. 10.1083/jcb.200204076

*We revealed that a member of dystrophin complex called alpha1-syntrophin plays an important role in regeneration of skeletal muscle and neuromuscular junctions.

2. Sakamoto M, Yuasa K, Yoshimura M, Yokota T, Ikemoto T, Suzuki M, Dickson G, Miyagoe-Suzuki Y, Takeda S. 

Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene, 

Biochem Biophys Res Commun. 2002, 293:1265-1272. 10.1016/S0006-291X(02)00362-5

—2000— 

1. Yokota T, Hosaka Y, Tsukita K, Kameya S, Shibuya S, Matsuda R, Wakayama Y, Takeda S. 

Aquaporin-4 is absent at the sarcolemma and at perivascular astrocyte endfeet in alpha1-syntrophin knockout mice, 

Proc Jpn Acad. 2000, 76B:22-27. https://doi.org/10.2183/pjab.76.22

*We revealed that a member of dystrophin complex called alpha1-syntrophin is responsible for membrane localization of a water channel aquaporin-4 in muscle and brain.

b) Preprints

8. Wilton-Clark H*, Yan E*, Yokota T.

Preparing for patient-customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases.

Preprints 2024,2024051955 (doi: 10.20944/preprints202405.1955.v1).

7. Leckie J*, Yokota T.

Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA.

Preprints 2024,2024050274 (doi: 10.20944/preprints202405.0274.v1).

6. Wilton-Clark H*, Al-aghbari A*, Yang J, Yokota T. 

Advancing Epidemiology and Genetic Approaches for the Treatment of Spinal and Bulbar Muscular Atrophy (SBMA): Focus on Prevalence in the Indigenous Population of Western Canada

Preprints 2023,2023071409. https://doi.org/10.20944/preprints202307.1409.v1

5. Zhu A*, Chiba S, Shimizu Y, Kunitake K, Okuno Y, Aoki Y, Yokota T

Use of 3-Way Voting of Machine Learning Algorithms Improves Prediction Performance of the Efficacy of Antisense-Mediated Exon Skipping and Reduces the Computational Burden

Preprints 2023, 2023030167. https://doi.org/10.20944/preprints202303.0167.v1

4. Wilton-Clark H*, Yokota T. 

Recent Trends in Antisense Therapies for Duchenne muscular dystrophy. 

Preprints 2023, 2023010139 (doi: 10.20944/preprints202301.0139.v1).

3. Lim KRQ*, Nguyen Q*, Dzierlega K*, Huang Y*, Yokota T. 

CRISPR-generated animal models of Duchenne muscular dystrophy. 

Preprints 2020, 2020030048. 10.3390/genes11030342

2. Nguyen Q*, Lim KRQ*, Yokota T. 

Genome Editing for the Understanding and Treatment of Cardiomyopathies. 

Preprints 2019, 2019120385 (doi: 10.20944/preprints201912. 0385.v1).

1. Lim KRQ*, Yoon C*, Yokota T. 

Methods of CRISPR/Cas9 Exon Skipping for Duchenne Muscular Dystrophy. 

Preprints 2018, 2018110018 (doi: 10.20944/preprints201811. 0018.v1).

c) Book Chapters

39. Sheri N, Yokota T.

In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice.

Skeletal Muscle Stem Cells: Methods Mol Biol. 2023, 327-336.

38. He M, Yokota T.

Exons 45–55 Skipping Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells.

Skeletal Muscle Stem Cells: Methods Mol Biol. 2023, 313-325.

37. Anwar S*, Yokota T.

Morpholino-mediated exons 28-29 skipping of dysferlin and characterization of multiexon-skipped dysferlin using RT-PCR, immunoblotting, and membrane wounding assay. 

Muscular Dystrophy Therapeutics: Methods Mol Biol. 2023, 183-196.

36. Lim KRQ*, Yokota T.

Current strategies of muscular dystrophy therapeutics: An overview. 

Muscular Dystrophy Therapeutics: Methods Mol Biol. 2023, 3-30. 

35. Roshmi RR*, Yokota T.

Viltolarsen: From preclinical studies to FDA approval. 

Muscular Dystrophy Therapeutics: Methods Mol Biol. 2023, 31-41.

34. Echigoya Y*, Yokota T.

Restoring dystrophin expression with Exon 44 and 53 skipping in the DMD gene in immortalized myotubes. 

Muscular Dystrophy Therapeutics: Methods Mol Biol. 2023, 125-139.

33. Shah MNA*, Yokota T. 

Restoring dystrophin expression by skipping exons 6 and 8 in neonatal dystrophic dogs. 

Muscular Dystrophy Therapeutics: Methods Mol Biol. 2023, 107-124.

32. Anwar S*, Yokota T. 

Rapid freezing of skeletal and cardiac muscles using isopentane cooled with liquid nitrogen and tragacanth gum for histological, genetic, and protein expression studies. 

Muscular Dystrophy Therapeutics: Methods Mol Biol.  2023, 45-53.

31. Lim KRQ*, Yokota T. 

Knocking down DUX4 in immortalized facioscapulohumeral muscular dystrophy patient-derived muscle cells. 

Muscular Dystrophy Therapeutics: Methods Mol Biol. 2023, 197-208

30. Maruyama R*, Yokota T.  

Antisense Oligonucleotide Treatment in a Humanized Mouse Model of Duchenne Muscular Dystrophy and Highly Sensitive Detection of Dystrophin Using Western Blotting.

Mouse Genetics: Methods Mol Biol. 2021, 2224:203-14. 

29. Chan L*, Yokota T. 

Development and Clinical Applications of Antisense Oligonucleotide Gapmers. 

Gapmers: Methods Mol Biol. 2020, 2176:21-47.  

28. Maruyama R*, Yokota T. 

Knocking Down Long Noncoding RNAs Using Antisense Oligonucleotide Gapmers. 

Gapmers: Methods Mol Biol. 2020, 2176:49-56. 

27. Lim K*, Yokota T. 

Detection of locked nucleic acid gapmers from mouse samples using ELISA. 

Gapmers: Methods Mol Biol. 2020, 2176:165-171.

26. Nguyen Q*, Yokota T. 

Degradation of toxic RNA in myotonic dystrophy using gapmer antisense oligonucleotides. 

Gapmers: Methods Mol Biol. 2020, 2176:99-109. 

25. Mahfouz M*, Maruyama R*, Yokota T. 

Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis. 

Gapmers: Methods Mol Biol. 2020, 2176:87-98.

24. Aslesh T*, Yokota T. 

Development of gapmer antisense oligonucleotides for genetic dyslipidemia and lipodystrophy. 

Gapmers: Methods Mol Biol. 2020, 2176:69-85.

23. Aslesh T*, Yokota T. 

Development of gapmer antisense oligonucleotides for Huntington's disease. 

Gapmers: Methods Mol Biol. 2020, 2176:57-67.

22. Lim K*, Yokota T. 

Invention and early history of gapmers. 

Gapmers: Methods Mol Biol. 2020, 2176:3-19.

21. Goodkey K*, Aslesh T*, Maruyama R*, Yokota T. 

Nusinersen in the treatment of spinal muscular atrophy. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:69-76. 

20. Touznik A*, Maruyama R*, Yokota T. 

In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 2828:439-454. 

19. Rodrigues M*, Yokota T. 

An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:31-55. 

18. Lim K*, Yokota T. 

Invention and early history of exon skipping and splice modulation. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:3-30. 

17. Maruyama R*, Yokota T. 

Tips to design splice-switching oligonucleotides for exon skipping and exon inclusion. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:79-90. 

16. Hara Y, Mizobe Y, Miyatake S, Takizawa H, Nagata T, Yokota T, Takeda S, Aoki Y. 

Exon skipping using antisense oligonucleotides for laminin-alpha2-deficient muscular dystrophy. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:553-564.

15. Mizobe Y, Miyatake S, Takizawa H, Hara Y, Yokota T, Nakamura A, Takeda S, Aoki Y. 

In vivo evaluation of single- and multi-exon skipping in mdx52 mice. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:275-292.

14. Melo D*, Yokota T. 

Systemic injection of peptide-PMOs and evaluation by RT-PCR and ELISA. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:263-273.

13. Maruyama R*, Aoki Y, Takeda S, Yokota T.

In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:365-379. 

*Cover image selection

12. Son H*, Yokota T. 

Recent advances and clinical applications of exon inclusion for SMA. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:57-68. 10.1007/978-1-4939-8651-4_3

11. Maruyama R*, Yokota T. 

Morpholino-mediated exon skipping targeting human ACVR1/ALK2 for fibrodysplasia ossificans progressiva. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:497-502. 10.1007/978-1-4939-8651-4_32

10. Lim K*, Yokota T. 

Quantitative evaluation of exon skipping in immortalized muscle cells in vitro. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:127-139. 10.1007/978-1-4939-8651-4_7

9. Aslesh T*, Maruyama R*, Yokota T. 

Systemic and ICV injections of antisense oligos into SMA mice and evaluation. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:455-465. 10.1007/978-1-4939-8651-4_28

8. Lee JJA*, Saito T, Duddy W, Takeda S, Yokota T. 

Direct reprogramming of human DMD fibroblasts into myotubes for in vitro evaluation of antisense-mediated exon skipping and exons 45-55 skipping accompanied by rescue of dystrophin expression. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:141-150. 10.1007/978-1-4939-8651-4_8

7. Maruyama R*, Yokota T. 

Creation of DMD muscle cell model using CRISPR-Cas9 genome editing to test the efficacy of exon skipping. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:165-171. 10.1007/978-1-4939-8651-4_10

6. Nakamura A, Aoki Y, Tsoumpra M, Yokota T, Takeda S. 

In vitro Multi-exon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient. 

Exon Skipping and Inclusion Therapies: Methods Mol Biol. 2018, 1828:151-163.  

5. Miyatake S, Mizobe Y, Takizawa H, Hara Y, Yokota T, Takeda S, Aoki Y. 

Exon skipping therapy using phosphorodiamidate morpholino oligomers in the mdx52 mouse model of Duchenne muscular dystrophy. 

Duchenne Muscular Dystrophy: Methods Mol Biol. 2018, 1687:123-141. 

4. Shimo T*, Maruyama R*, Yokota T. 

Designing effective antisense oligonucleotides for exon skipping. 

Duchenne Muscular Dystrophy: Methods Mol Biol. 2018, 1687:143-155.

3. Maruyama R*, Echigoya Y*, Caluseriu O, Aoki Y, Takeda S, Yokota T. 

Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy. 

Morpholino Oligomers: Methods Mol Biol.2017, 1565:201-213. 10.1007/978-1-4939-6817-6_17

2. Lee JJA*, Yokota T. 

Translational research in nucleic acid therapies for muscular dystrophies. 

Translational Research in Muscular Dystrophy (pp. 87-102), 2016, © Springer., Tokyo, Japan 

1. Yokota T* (corresponding), Hoffman E, Takeda S. 

Antisense oligo-mediated multi-exon-skipping in a dog model of Duchenne muscular dystrophy. 

Muscle Gene Therapy: Methods Mol Biol. 2011, 709;299-312. 

d) Books 

3. Yokota T and Maruyama R (Eds). 

Muscular Dystrophy Therapeutics

Methods in Molecular Biology, 2023 Springer-Nature

2. Yokota T and Maruyama R (Eds). 

Gapmers

Methods in Molecular Biology, 2020 Springer-Nature

1. Yokota T and Maruyama R (Eds). 

Exon Skipping and Inclusion Therapies

Methods in Molecular Biology, 2018, Springer-Nature

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Last updated: 12 June 2024