Zobrazeno 1 - 10
of 50
pro vyhledávání: '"Yusuke Echigoya"'
Publikováno v:
International Journal of Molecular Sciences, Vol 24, Iss 19, p 14846 (2023)
RNase H-dependent gapmer antisense oligonucleotides (ASOs) are a promising therapeutic approach via sequence-specific binding to and degrading target RNAs. However, the efficacy and mechanism of antiviral gapmer ASOs have remained unclear. Here, we i
Externí odkaz:
https://doaj.org/article/a841da77908d4b93a2cc028e41a10c14
Autor:
Yusuke Echigoya, Nhu Trieu, William Duddy, Hong M. Moulton, HaiFang Yin, Terence A. Partridge, Eric P. Hoffman, Joe N. Kornegay, Frank A. Rohret, Christopher S. Rogers, Toshifumi Yokota
Publikováno v:
International Journal of Molecular Sciences, Vol 22, Iss 23, p 13065 (2021)
Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive disorder caused by mutations in the DMD gene and the subsequent lack of dystrophin protein. Recently, phosphorodiamidate morpholino oligomer (PMO)-antisense oligonucleotides (ASOs) targ
Externí odkaz:
https://doaj.org/article/8db7c319f66e47b79bb85b25a5d9eb29
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-9 (2017)
Abstract Spinal muscular atrophy (SMA) is an autosomal recessive disorder affecting motor neurons, and is currently the most frequent genetic cause of infant mortality. SMA is caused by a loss-of-function mutation in the survival motor neuron 1 (SMN1
Externí odkaz:
https://doaj.org/article/fe21411861714e70b228c74f8aefdfe3
Autor:
Joshua Lee, Yusuke Echigoya, William Duddy, Takashi Saito, Yoshitsugu Aoki, Shin'ichi Takeda, Toshifumi Yokota
Publikováno v:
PLoS ONE, Vol 13, Iss 5, p e0197084 (2018)
Antisense-mediated exon skipping has made significant progress as a therapeutic platform in recent years, especially in the case of Duchenne muscular dystrophy (DMD). Despite FDA approval of eteplirsen-the first-ever antisense drug clinically markete
Externí odkaz:
https://doaj.org/article/0b8656d1378e49f18266964da5844ba6
Publikováno v:
Journal of Personalized Medicine, Vol 8, Iss 4, p 41 (2018)
Duchenne muscular dystrophy (DMD), a fatal X-linked recessive disorder, is caused mostly by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense oligonucleotide-mediated exon skipping is a promising therapy for DMD. Exon s
Externí odkaz:
https://doaj.org/article/11f0ed2f8fb5487d97caaa3c3f0dbe49
Autor:
Yusuke Echigoya, Yoshitsugu Aoki, Bailey Miskew, Dharminder Panesar, Aleksander Touznik, Tetsuya Nagata, Jun Tanihata, Akinori Nakamura, Kanneboyina Nagaraju, Toshifumi Yokota
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 4, Iss C (2015)
Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining challenges include the limited applicability to patients and unclear function of truncated dyst
Externí odkaz:
https://doaj.org/article/6bc18960718c4289bda5d2ae2bc32605
Publikováno v:
PLoS ONE, Vol 10, Iss 3, p e0120058 (2015)
The use of antisense 'splice-switching' oligonucleotides to induce exon skipping represents a potential therapeutic approach to various human genetic diseases. It has achieved greatest maturity in exon skipping of the dystrophin transcript in Duchenn
Externí odkaz:
https://doaj.org/article/eafa499c76f74bc48dc9702912a13a4e
Autor:
Yusuke Echigoya, Joshua Lee, Merryl Rodrigues, Tetsuya Nagata, Jun Tanihata, Ashkan Nozohourmehrabad, Dharminder Panesar, Bailey Miskew, Yoshitsugu Aoki, Toshifumi Yokota
Publikováno v:
PLoS ONE, Vol 8, Iss 7, p e69194 (2013)
Duchenne muscular dystrophy (DMD), one of the most common and lethal genetic disorders, and the mdx mouse myopathies are caused by a lack of dystrophin protein. These dystrophic muscles contain sporadic clusters of dystrophin-expressing revertant fib
Externí odkaz:
https://doaj.org/article/1ddac4de1d0e4d3182a09b6404df8041
Autor:
Yusuke Echigoya, Toshifumi Yokota
Publikováno v:
Methods in Molecular Biology ISBN: 9781071627716
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::8f1bbaa3af39a23f979a5bf859d2e07f
https://doi.org/10.1007/978-1-0716-2772-3_7
https://doi.org/10.1007/978-1-0716-2772-3_7
Autor:
Yusuke, Echigoya, Toshifumi, Yokota
Publikováno v:
Methods in molecular biology (Clifton, N.J.). 2587
Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is a therapeutic approach that applies to many Duchenne muscular dystrophy (DMD) patients harboring out-of-frame deletion mutations in the DMD gene. In particular, PMOs for skipping