Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5

Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5

Alport syndrome is a progressive inherited nephritis accompanied by sensorineural loss of hearing and ocular abnormalities, for which there is currently no effective therapy. Here, the authors develop an exon-skipping therapy using an antisense-oligonucleotide and show it is effective in mouse model...

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Autores principales: Tomohiko Yamamura, Tomoko Horinouchi, Tomomi Adachi, Maki Terakawa, Yutaka Takaoka, Kohei Omachi, Minoru Takasato, Kiyosumi Takaishi, Takao Shoji, Yoshiyuki Onishi, Yoshito Kanazawa, Makoto Koizumi, Yasuko Tomono, Aki Sugano, Akemi Shono, Shogo Minamikawa, China Nagano, Nana Sakakibara, Shinya Ishiko, Yuya Aoto, Misato Kamura, Yutaka Harita, Kenichiro Miura, Shoichiro Kanda, Naoya Morisada, Rini Rossanti, Ming Juan Ye, Yoshimi Nozu, Masafumi Matsuo, Hirofumi Kai, Kazumoto Iijima, Kandai Nozu
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Lenguaje:EN
Publicado: Nature Portfolio 2020
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Acceso en línea:https://doaj.org/article/165df2dbd3f44073aaae13fc966489b5
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spelling oai:doaj.org-article:165df2dbd3f44073aaae13fc966489b52021-12-02T15:02:55ZDevelopment of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A510.1038/s41467-020-16605-x2041-1723https://doaj.org/article/165df2dbd3f44073aaae13fc966489b52020-06-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-16605-xhttps://doaj.org/toc/2041-1723Alport syndrome is a progressive inherited nephritis accompanied by sensorineural loss of hearing and ocular abnormalities, for which there is currently no effective therapy. Here, the authors develop an exon-skipping therapy using an antisense-oligonucleotide and show it is effective in mouse models.Tomohiko YamamuraTomoko HorinouchiTomomi AdachiMaki TerakawaYutaka TakaokaKohei OmachiMinoru TakasatoKiyosumi TakaishiTakao ShojiYoshiyuki OnishiYoshito KanazawaMakoto KoizumiYasuko TomonoAki SuganoAkemi ShonoShogo MinamikawaChina NaganoNana SakakibaraShinya IshikoYuya AotoMisato KamuraYutaka HaritaKenichiro MiuraShoichiro KandaNaoya MorisadaRini RossantiMing Juan YeYoshimi NozuMasafumi MatsuoHirofumi KaiKazumoto IijimaKandai NozuNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-8 (2020)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Tomohiko Yamamura
Tomoko Horinouchi
Tomomi Adachi
Maki Terakawa
Yutaka Takaoka
Kohei Omachi
Minoru Takasato
Kiyosumi Takaishi
Takao Shoji
Yoshiyuki Onishi
Yoshito Kanazawa
Makoto Koizumi
Yasuko Tomono
Aki Sugano
Akemi Shono
Shogo Minamikawa
China Nagano
Nana Sakakibara
Shinya Ishiko
Yuya Aoto
Misato Kamura
Yutaka Harita
Kenichiro Miura
Shoichiro Kanda
Naoya Morisada
Rini Rossanti
Ming Juan Ye
Yoshimi Nozu
Masafumi Matsuo
Hirofumi Kai
Kazumoto Iijima
Kandai Nozu
Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
description Alport syndrome is a progressive inherited nephritis accompanied by sensorineural loss of hearing and ocular abnormalities, for which there is currently no effective therapy. Here, the authors develop an exon-skipping therapy using an antisense-oligonucleotide and show it is effective in mouse models.
format article
author Tomohiko Yamamura
Tomoko Horinouchi
Tomomi Adachi
Maki Terakawa
Yutaka Takaoka
Kohei Omachi
Minoru Takasato
Kiyosumi Takaishi
Takao Shoji
Yoshiyuki Onishi
Yoshito Kanazawa
Makoto Koizumi
Yasuko Tomono
Aki Sugano
Akemi Shono
Shogo Minamikawa
China Nagano
Nana Sakakibara
Shinya Ishiko
Yuya Aoto
Misato Kamura
Yutaka Harita
Kenichiro Miura
Shoichiro Kanda
Naoya Morisada
Rini Rossanti
Ming Juan Ye
Yoshimi Nozu
Masafumi Matsuo
Hirofumi Kai
Kazumoto Iijima
Kandai Nozu
author_facet Tomohiko Yamamura
Tomoko Horinouchi
Tomomi Adachi
Maki Terakawa
Yutaka Takaoka
Kohei Omachi
Minoru Takasato
Kiyosumi Takaishi
Takao Shoji
Yoshiyuki Onishi
Yoshito Kanazawa
Makoto Koizumi
Yasuko Tomono
Aki Sugano
Akemi Shono
Shogo Minamikawa
China Nagano
Nana Sakakibara
Shinya Ishiko
Yuya Aoto
Misato Kamura
Yutaka Harita
Kenichiro Miura
Shoichiro Kanda
Naoya Morisada
Rini Rossanti
Ming Juan Ye
Yoshimi Nozu
Masafumi Matsuo
Hirofumi Kai
Kazumoto Iijima
Kandai Nozu
author_sort Tomohiko Yamamura
title Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
title_short Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
title_full Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
title_fullStr Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
title_full_unstemmed Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
title_sort development of an exon skipping therapy for x-linked alport syndrome with truncating variants in col4a5
publisher Nature Portfolio
publishDate 2020
url https://doaj.org/article/165df2dbd3f44073aaae13fc966489b5
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