A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
经翻译优化的AAV-UGT1A1载体可安全、持久地矫正克里格勒-纳贾尔综合征
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1038/mtm.2016.49
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