Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Cukras, Catherine ; Wiley, Henry E. ; Jeffrey, Brett G. ; Sen, H. Nida ; Turriff, Amy ; Zeng, Yong ; Vijayasarathy, Camasamudram ; Marangoni, Dario ; Ziccardi, Lucia ; Kjellstrom, Sten ; Park, Tae Kwon ; Hiriyanna, Suja ; Wright, J. Fraser ; Colosi, Peter ; Wu, Zhijian ; Bush, Ronald A. ; Wei, Lisa L. ; Sieving, Paul A.

Molecular therapy, 2018-09, Vol.26 (9), p.2282-2294 [Periódico revisado por pares]

United States: Elsevier Inc

Texto completo disponível