Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model von Hua, Yimin, Sahashi, Kentaro, Rigo, Frank, Hung, Gene, Horev, Guy, Bennett, C. Frank, Krainer, Adrian R.

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Preclinical progression of neurodegenerative diseases von Katsuno, Masahisa, Sahashi, Kentaro, Iguchi, Yohei, Hashizume, Atsushi

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Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models von Hua, Yimin, Liu, Ying Hsiu, Sahashi, Kentaro, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R

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Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model von Hua, Yimin, Sahashi, Kentaro, Hung, Gene, Rigo, Frank, Passini, Marco A, Bennett, C Frank, Krainer, Adrian R

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Mid1 is associated with androgen-dependent axonal vulnerability of motor neurons in spinal and bulbar muscular atrophy von Ogura, Yosuke, Sahashi, Kentaro, Hirunagi, Tomoki, Iida, Madoka, Miyata, Takaki, Katsuno, Masahisa

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Neutrophil extracellular traps: from antimicrobial innate immunity to the development of chemotherapy-induced peripheral neuropathy von Koike, Haruki, Iguchi, Yohei, Sahashi, Kentaro, Katsuno, Masahisa

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Nucleic Acid-Based Therapeutic Approach for Spinal and Bulbar Muscular Atrophy and Related Neurological Disorders von Hirunagi, Tomoki, Sahashi, Kentaro, Meilleur, Katherine G, Katsuno, Masahisa

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Exercise attenuates polyglutamine‐mediated neuromuscular degeneration in a mouse model of spinal and bulbar muscular atrophy von Hirunagi, Tomoki, Nakatsuji, Hideaki, Sahashi, Kentaro, Yamamoto, Mikiyasu, Iida, Madoka, Tohnai, Genki, Kondo, Naohide, Yamada, Shinichiro, Murakami, Ayuka, Noda, Seiya, Adachi, Hiroaki, Sobue, Gen, Katsuno, Masahisa

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BIIB021, a synthetic Hsp90 inhibitor, induces mutant ataxin-1 degradation through the activation of heat shock factor 1 von Ding, Ying, Adachi, Hiroaki, Katsuno, Masahisa, Sahashi, Kentaro, Kondo, Naohide, Iida, Madoka, Tohnai, Genki, Nakatsuji, Hideaki, Sobue, Gen

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Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS von Hirunagi, Tomoki, Sahashi, Kentaro, Tachikawa, Kiyoshi, Leu, Angel I., Nguyen, Michelle, Mukthavaram, Rajesh, Karmali, Priya P., Chivukula, Padmanabh, Tohnai, Genki, Iida, Madoka, Onodera, Kazunari, Ohyama, Manabu, Okada, Yohei, Okano, Hideyuki, Katsuno, Masahisa

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HMGCR antibody-associated myopathy as a paraneoplastic manifestation of esophageal carcinoma von Tsujikawa, Koyo, Hara, Kazuhiro, Muro, Yoshinao, Nakanishi, Hirotaka, Niwa, Yukiko, Koike, Masahiko, Noda, Seiya, Riku, Yuichi, Sahashi, Kentaro, Atsuta, Naoki, Ito, Mizuki, Shimoyama, Yoshie, Akiyama, Masashi, Katsuno, Masahisa

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Actin-binding protein filamin-A drives tau aggregation and contributes to progressive supranuclear palsy pathology von Tsujikawa, Koyo, Hamanaka, Kohei, Riku, Yuichi, Hattori, Yuki, Hara, Norikazu, Iguchi, Yohei, Ishigaki, Shinsuke, Hashizume, Atsushi, Miyatake, Satoko, Mitsuhashi, Satomi, Miyazaki, Yu, Kataoka, Mayumi, Jiayi, Li, Yasui, Keizo, Kuru, Satoshi, Koike, Haruki, Kobayashi, Kenta, Sahara, Naruhiko, Ozaki, Norio, Yoshida, Mari, Kakita, Akiyoshi, Saito, Yuko, Iwasaki, Yasushi, Miyashita, Akinori, Iwatsubo, Takeshi, Ikeuchi, Takeshi, Miyata, Takaki, Sobue, Gen, Matsumoto, Naomichi, Sahashi, Kentaro, Katsuno, Masahisa

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Pathological impact of SMN2 mis‐splicing in adult SMA mice von Sahashi, Kentaro, Ling, Karen K. Y., Hua, Yimin, Wilkinson, John Erby, Nomakuchi, Tomoki, Rigo, Frank, Hung, Gene, Xu, David, Jiang, Ya‐Ping, Lin, Richard Z., Ko, Chien‐Ping, Bennett, C. Frank, Krainer, Adrian R.

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In vitro and in silico analysis reveals an efficient algorithm to predict the splicing consequences of mutations at the 5' splice sites von Sahashi, Kentaro, Masuda, Akio, Matsuura, Tohru, Shinmi, Jun, Zhang, Zhujun, Takeshima, Yasuhiro, Matsuo, Masafumi, Sobue, Gen, Ohno, Kinji

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Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice von Sinha, Rahul, Kim, Young Jin, Nomakuchi, Tomoki, Sahashi, Kentaro, Hua, Yimin, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R

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NUS1 mutation in a family with epilepsy, cerebellar ataxia, and tremor von Araki, Kunihiko, Nakamura, Ryoichi, Ito, Daisuke, Kato, Kohji, Iguchi, Yohei, Sahashi, Kentaro, Toyama, Miho, Hamada, Kensuke, Okamoto, Nobuhiko, Wada, Yoshinao, Nakamura, Tomohiko, Ogi, Tomoo, Katsuno, Masahisa

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The SYNGAP1 3'UTR Variant in ALS Patients Causes Aberrant SYNGAP1 Splicing and Dendritic Spine Loss by Recruiting HNRNPK von Yokoi, Satoshi, Ito, Takuji, Sahashi, Kentaro, Nakatochi, Masahiro, Nakamura, Ryoichi, Tohnai, Genki, Fujioka, Yusuke, Ishigaki, Shinsuke, Udagawa, Tsuyoshi, Izumi, Yuishin, Morita, Mitsuya, Kano, Osamu, Oda, Masaya, Sone, Takefumi, Okano, Hideyuki, Atsuta, Naoki, Katsuno, Masahisa, Okada, Yohei, Sobue, Gen

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Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy von Sahashi, Kentaro, Katsuno, Masahisa, Hung, Gene, Adachi, Hiroaki, Kondo, Naohide, Nakatsuji, Hideaki, Tohnai, Genki, Iida, Madoka, Bennett, C Frank, Sobue, Gen

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Antisense therapy of spinal muscular atrophy ―beyond the achievement von Sahashi, Kentaro, Katsuno, Masahisa

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V. Spinal and Bulbar Muscular Atrophy (SBMA) and Bulbar Muscular Atrophy (SMA) von Yamada, Shinichiro, Hirunagi, Tomoki, Sahashi, Kentaro, Katsuno, Masahisa

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