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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Vydáno v:Nat Commun
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group UK 2020
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Article
On-line přístup: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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