A translationally optimized AAV-UGT1A1 vector drives safe...

Molecular Therapy — Methods & Clinical Development 2016 / 7 Vol. 3

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, L
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Volume:
3
Language:
english
Journal:
Molecular Therapy — Methods & Clinical Development
DOI:
10.1038/mtm.2016.49
Date:
July, 2016
File:
PDF, 1.11 MB
english, 2016
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