Zobrazeno 1 - 10
of 224
pro vyhledávání: '"Toshifumi, Yokota"'
Publikováno v:
Current Research in Toxicology, Vol 7, Iss , Pp 100182- (2024)
Duchenne Muscular Dystrophy (DMD) is a devastating X-linked genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. This results in the absence or dysfunction of the dystrophin protein, leading to mu
Externí odkaz:
https://doaj.org/article/4bc8b59de5f7483da8fd0386aaf8f26c
Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA
Autor:
Jamie Leckie, Toshifumi Yokota
Publikováno v:
Molecules, Vol 29, Iss 11, p 2658 (2024)
Spinal muscular atrophy (SMA) is a severe neuromuscular disorder that is caused by mutations in the survival motor neuron 1 (SMN1) gene, hindering the production of functional survival motor neuron (SMN) proteins. Antisense oligonucleotides (ASOs), a
Externí odkaz:
https://doaj.org/article/d0ec8c78291249d482c26911d5c36e33
Autor:
Md Nur Ahad Shah, Toshifumi Yokota
Publikováno v:
Therapeutic Advances in Neurological Disorders, Vol 16 (2023)
Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting complications such as loss of skeletal muscle function as well as respiratory and cardiac complications. Advanced therapeutics in pulmonary care have significant
Externí odkaz:
https://doaj.org/article/78bba276c0f6425cb4af4d231dd034c3
Autor:
Saeed Anwar, Toshifumi Yokota
Publikováno v:
Biomolecules, Vol 14, Iss 3, p 256 (2024)
Dysferlinopathies refer to a spectrum of muscular dystrophies that cause progressive muscle weakness and degeneration. They are caused by mutations in the DYSF gene, which encodes the dysferlin protein that is crucial for repairing muscle membranes.
Externí odkaz:
https://doaj.org/article/ed09bdbaeef84aada5e86706aad96696
Autor:
Zorica Nakevska, Toshifumi Yokota
Publikováno v:
European Journal of Cell Biology, Vol 102, Iss 2, Pp 151326- (2023)
Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness. SMN1 normally produces an e
Externí odkaz:
https://doaj.org/article/83e7a8b53aa34556a3cbb1b87ca79547
Autor:
Harry Wilton-Clark, Toshifumi Yokota
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 500-501 (2023)
Externí odkaz:
https://doaj.org/article/090d09047714444f8c25549ad0f1e894
Autor:
Umme Sabrina Haque, Toshifumi Yokota
Publikováno v:
Cells, Vol 12, Iss 19, p 2395 (2023)
Antisense oligonucleotide-based (ASO) therapeutics have emerged as a promising strategy for the treatment of human disorders. Charge-neutral PMOs have promising biological and pharmacological properties for antisense applications. Despite their great
Externí odkaz:
https://doaj.org/article/6d0c5b7f4f3942d896675df2cc607cf6
Autor:
Tejal Aslesh, Esra Erkut, Jun Ren, Kenji Rowel Q. Lim, Stanley Woo, Susan Hatlevig, Hong M. Moulton, Simon Gosgnach, John Greer, Rika Maruyama, Toshifumi Yokota
Publikováno v:
JCI Insight, Vol 8, Iss 5 (2023)
Antisense oligonucleotide–mediated (AO-mediated) therapy is a promising strategy to treat several neurological diseases, including spinal muscular atrophy (SMA). However, limited delivery to the CNS with AOs administered intravenously or subcutaneo
Externí odkaz:
https://doaj.org/article/4bc8603502624b95ab09f5a97da8d198
Publikováno v:
Frontiers in Genome Editing, Vol 5 (2023)
Externí odkaz:
https://doaj.org/article/1ebe4b5124dd4e9db367cd2570b2429b
Autor:
Alex Zhu, Shuntaro Chiba, Yuki Shimizu, Katsuhiko Kunitake, Yasushi Okuno, Yoshitsugu Aoki, Toshifumi Yokota
Publikováno v:
Pharmaceutics, Vol 15, Iss 7, p 1808 (2023)
Antisense oligonucleotide (ASO)-mediated exon skipping has become a valuable tool for investigating gene function and developing gene therapy. Machine-learning-based computational methods, such as eSkip-Finder, have been developed to predict the effi
Externí odkaz:
https://doaj.org/article/f28c248aea3b4f4b936f9abb21a44cd7