Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2
通过直接向脑实质注射表达 CLN2 的 rh.10 腺相关病毒来减缓晚期婴儿巴顿病
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.abb5413
Dolan Sondhi, Stephen M Kaminsky, Neil R Hackett, Odelya E Pagovich, Jonathan B Rosenberg, Bishnu P De, Alvin Chen, Benjamin Van de Graaf, Jason G Mezey, Grace W Mammen, Denesy Mancenido, Fang Xu, Barry Kosofsky, Kaleb Yohay, Stefan Worgall, Robert J Kaner, Mark Souwedaine, Bruce M Greenwald, Michae