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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Published in:Mol Ther Methods Clin Dev
Main Authors: 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
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2018
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6348934/
https://ncbi.nlm.nih.gov/pubmed/30705921
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.12.011
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