A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Titel:
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Auteur:
Ronzitti, Giuseppe Bortolussi, Giulia van Dijk, Remco Collaud, Fanny Charles, Severine Leborgne, Christian Vidal, Patrice Martin, Samia Gjata, Bernard Sola, Marcelo Simon van Wittenberghe, Laetitia Vignaud, Alban Veron, Philippe Bosma, Piter J Muro, Andres F Mingozzi, Federico
Verschenen in:
Molecular therapy. Methods & clinical development
Paginering:
Jaargang 3 (2016) nr. C pagina's 1 p.
Jaar:
2016
Inhoud:
Uitgever:
Official journal of the American Society of Gene & Cell Therapy