AAV-delivered PPT1 provides long-term neurological benefits in CLN1 mice and achieves therapeutic levels in sheep brain
AAV递送的PPT1在CLN1小鼠中可提供长期的神经系统获益，并在绵羊脑中达到治疗水平。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.07.011
Alam, Md Suhail; Khatiwada, Apeksha; Eaton, Samantha L; Cohen, Daniel M; White, John; Derby, Melissa; Peterson, Drew; Rivera-Pena, Graciela; Nayal, Mohamad; Becker, Mallory; Beck, Heena; Li, Charlie; Gentzel, Renee; Atkins, George; Greenhalgh, Stephen N; Lillico, Simon G; Gregson, Rachael; Clutton, Eddie; Murdoch, Fraser; Nixon, James; Gray, Mark; Thompson, Gerard; McBride, Jodi; Wishart, Thomas M; Biferi, Maria Grazia; Ramsburg, Elizabeth
