Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis

Complement-mediated damage to the neuromuscular junction (NMJ) is a key mechanism of pathology in myasthenia gravis (MG), and therapeutics inhibiting complement have shown evidence of efficacy in the treatment of MG. In this study, we describe the development of a subcutaneously administered N-acety...

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Detalles Bibliográficos
Autores principales: Kusner, Linda L., Yucius, Kristina, Sengupta, Manjistha, Sprague, Andrew G., Desai, Dhruv, Nguyen, Tuyen, Charisse, Klaus, Kuchimanchi, Satya, Kallanthottathil, Rajeev, Fitzgerald, Kevin, Kaminski, Henry J., Borodovsky, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6539425/
https://www.ncbi.nlm.nih.gov/pubmed/31193726
http://dx.doi.org/10.1016/j.omtm.2019.04.009

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