Reversal of neuroinflammation in novel GS model mice by single i.c.v. administration of CHO-derived rhCTSA precursor protein

Galactosialidosis (GS) is a lysosomal cathepsin A (CTSA) deficiency. It associates with a simultaneous decrease of neuraminidase 1 (NEU1) activity and sialylglycan storage. Central nervous system (CNS) symptoms reduce the quality of life of juvenile/adult-type GS patients, but there is no effective...

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Detalles Bibliográficos
Autores principales: Horii, Yuto, Iniwa, Toshiki, Onitsuka, Masayoshi, Tsukimoto, Jun, Tanaka, Yuki, Ike, Hironobu, Fukushi, Yuri, Ando, Haruna, Takeuchi, Yoshie, Nishioka, So-ichiro, Tsuji, Daisuke, Ikuo, Mariko, Yamazaki, Naoshi, Takiguchi, Yoshiharu, Ishimaru, Naozumi, Itoh, Kohji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9062439/
https://www.ncbi.nlm.nih.gov/pubmed/35573044
http://dx.doi.org/10.1016/j.omtm.2022.04.001

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