Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

Adeno-associated viruses (AAVs) are among the most efficient vectors for liver gene therapy. Results obtained in the first hemophilia clinical trials demonstrated the long-term efficacy of this approach in humans, showing efficient targeting of hepatocytes with both self-complementary (sc) and singl...

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Detalles Bibliográficos
Autores principales: Collaud, Fanny, Bortolussi, Giulia, Guianvarc’h, Laurence, Aronson, Sem J., Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine G., Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia v., Martin, Samia, Le Bec, Christine, Bosma, Piter J., Muro, Andres F., Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6348934/
https://www.ncbi.nlm.nih.gov/pubmed/30705921
http://dx.doi.org/10.1016/j.omtm.2018.12.011

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