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

Currently, there are no treatments for Alport syndrome, which is the second most commonly inherited kidney disease. Here we report the development of an exon-skipping therapy using an antisense-oligonucleotide (ASO) for severe male X-linked Alport syndrome (XLAS). We targeted truncating variants in...

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Detalhes bibliográficos
Publicado no:Nat Commun
Principais autores: Yamamura, Tomohiko, Horinouchi, Tomoko, Adachi, Tomomi, Terakawa, Maki, Takaoka, Yutaka, Omachi, Kohei, Takasato, Minoru, Takaishi, Kiyosumi, Shoji, Takao, Onishi, Yoshiyuki, Kanazawa, Yoshito, Koizumi, Makoto, Tomono, Yasuko, Sugano, Aki, Shono, Akemi, Minamikawa, Shogo, Nagano, China, Sakakibara, Nana, Ishiko, Shinya, Aoto, Yuya, Kamura, Misato, Harita, Yutaka, Miura, Kenichiro, Kanda, Shoichiro, Morisada, Naoya, Rossanti, Rini, Ye, Ming Juan, Nozu, Yoshimi, Matsuo, Masafumi, Kai, Hirofumi, Iijima, Kazumoto, Nozu, Kandai
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7265383/
https://ncbi.nlm.nih.gov/pubmed/32488001
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-16605-x
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