Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

Ornithine transcarbamylase deficiency (OTCD) is an X-linked liver disorder caused by partial or total loss of OTC enzyme activity. It is characterized by elevated plasma ammonia, leading to neurological impairments, coma, and death in the most severe cases. OTCD is managed by combining dietary restr...

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Detalles Bibliográficos
Autores principales: De Sabbata, Giulia, Boisgerault, Florence, Guarnaccia, Corrado, Iaconcig, Alessandra, Bortolussi, Giulia, Collaud, Fanny, Ronzitti, Giuseppe, Sola, Marcelo Simon, Vidal, Patrice, Rouillon, Jeremy, Charles, Severine, Nicastro, Emanuele, D’Antiga, Lorenzo, Ilyinskii, Petr, Mingozzi, Federico, Kishimoto, Takashi Kei, Muro, Andrés F.
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/PMC7786024/
https://www.ncbi.nlm.nih.gov/pubmed/33473356
http://dx.doi.org/10.1016/j.omtm.2020.11.005

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