biAb Mediated Restoration of the Linkage between Dystroglycan and Laminin-211 as a Therapeutic Approach for α-Dystroglycanopathies

Patients with α-dystroglycanopathies, a subgroup of rare congenital muscular dystrophies, present with a spectrum of clinical manifestations that includes muscular dystrophy as well as CNS and ocular abnormalities. Although patients with α-dystroglycanopathies are genetically heterogeneous, they sha...

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Detalles Bibliográficos
Autores principales: Gumlaw, Nathan, Sevigny, Leila M., Zhao, Hongmei, Luo, Zhengyu, Bangari, Dinesh S., Masterjohn, Elizabeth, Chen, Yangde, McDonald, Barbara, Magnay, Maureen, Travaline, Tara, Yoshida-Moriguchi, Takako, Fan, Wei, Reczek, David, Stefano, James E., Qiu, Huawei, Beil, Christian, Lange, Christian, Rao, Ercole, Lukason, Michael, Barry, Elizabeth, Brondyk, William H., Zhu, Yunxiang, Cheng, Seng H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7001080/
https://www.ncbi.nlm.nih.gov/pubmed/31843448
http://dx.doi.org/10.1016/j.ymthe.2019.11.023

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