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Degenerative Neurological and Neuromuscular Disease|April 17, 2025
Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging TherapiesUmme Sabrina Haque, Toshifumi YokotaGenes|August 29, 2024
Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and ChallengesUmme Sabrina Haque, Toshifumi YokotaCells|October 13, 2023
Enhancing Antisense Oligonucleotide-Based Therapeutic Delivery with DG9, a Versatile Cell-Penetrating PeptideUmme Sabrina Haque, Toshifumi YokotaMethods in Molecular Biology (Clifton, N.J.)|October 1, 2025
DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy MiceUmme Sabrina Haque, Melissa Kohut, Toshifumi YokotaCurrent Research in Toxicology|July 10, 2024
Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASOUmme Sabrina Haque, Melissa Kohut, Toshifumi YokotaFrontiers in Plant Science|February 6, 2023
Functional genomic analysis of K+ related salt-responsive transporters in tolerant and sensitive genotypes of riceUmme Sabrina Haque, Sabrina M Elias, Israt Jahan, et al.Molecular Therapy. Nucleic Acids|February 19, 2025
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cellsSaeed Anwar, Rohini Roy Roshmi, Stanley Woo, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|May 27, 2026
Lipid nanoparticle delivery of antisense gapmers attenuates pathology in a mouse model of facioscapulohumeral muscular dystrophySaeed Anwar, Karen Ying Tung Chan, Abdullah Zia, et al.Pageof 1