Gene therapy for Crigler-Najjar syndrome with AT342, a liver-targeted AAV8-UGT1A1 vector: preliminary results from a Phase 1/2 study (VALENS)S. Prasad, K. H. Strauss, P. McKiernan,F. Mavilio,G. Mazariegos,N. Ovchinsky, A. Dhawan,S. Ranganathan, M. Deheragoda,M. W. Lawlor,M. Noursalehi,W. P. KennedyHUMAN GENE THERAPY(2018)引用 0|浏览6暂无评分AI 理解论文溯源树样例生成溯源树,研究论文发展脉络Chat Paper正在生成论文摘要