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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7265383/
https://www.ncbi.nlm.nih.gov/pubmed/32488001
http://dx.doi.org/10.1038/s41467-020-16605-x

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