Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
用于治疗 Crigler-Najjar 综合征的 AAV8-hUGT1A1 载体的临床前开发
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2018.12.011
Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc'h, Sem J Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G Dufour, Florence Lacoste, Cyril Luc, Laetitia V Wittenberghe, Samia Martin, Christine Le Bec, Piter J Bosma, An