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...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Mol Ther Methods Clin Dev
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: American Society of Gene & Cell Therapy 2018
Θέματα:
Article
Διαθέσιμο Online: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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