A blocking monoclonal antibody reveals dimerization of intracellular domains of ALK2 associated with genetic disorders

por Katagiri, Takenobu, Tsukamoto, Sho, Kuratani, Mai, Tsuji, Shinnosuke, Nakamura, Kensuke, Ohte, Satoshi, Kawaguchi, Yoshiro, Takaishi, Kiyosumi
Publicado 2023

Publisher Correction: A blocking monoclonal antibody reveals dimerization of intracellular domains of ALK2 associated with genetic disorders

por Katagiri, Takenobu, Tsukamoto, Sho, Kuratani, Mai, Tsuji, Shinnosuke, Nakamura, Kensuke, Ohte, Satoshi, Kawaguchi, Yoshiro, Takaishi, Kiyosumi
Publicado 2023

Renadirsen, a Novel 2′OMeRNA/ENA(®) Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo

por Ito, Kentaro, Takakusa, Hideo, Kakuta, Masayo, Kanda, Akira, Takagi, Nana, Nagase, Hiroyuki, Watanabe, Nobuaki, Asano, Daigo, Goda, Ryoya, Masuda, Takeshi, Nakamura, Akifumi, Onishi, Yoshiyuki, Onoda, Toshio, Koizumi, Makoto, Takeshima, Yasuhiro, Matsuo, Masafumi, Takaishi, Kiyosumi
Publicado 2021

A splice-switching oligonucleotide treatment ameliorates glycogen storage disease type 1a in mice with G6PC c.648G>T

por Ito, Kentaro, Tajima, Go, Kamisato, Chikako, Tsumura, Miyuki, Iwamoto, Mitsuhiro, Sekiguchi, Yukiko, Numata, Yukinobu, Watanabe, Kyoko, Yabe, Yoshiyuki, Kanki, Satomi, Fujieda, Yusuke, Goto, Koichi, Sogawa, Yoshitaka, Oitate, Masataka, Nagase, Hiroyuki, Tsuji, Shinnosuke, Nishizawa, Tomohiro, Kakuta, Masayo, Masuda, Takeshi, Onishi, Yoshiyuki, Koizumi, Makoto, Nakamura, Hidefumi, Okada, Satoshi, Matsuo, Masafumi, Takaishi, Kiyosumi
Publicado 2023

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

por 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
Publicado 2020