参考文献:10.1007/s10545-011-9446-x 摘要:Roberts MS, Macauley SL, Wong AM, Yilmas D, Hohm S, Cooper JD, Sands MS. Combination small molecule PPT1 mimetic and CNS‐directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis. J of Inher Metab Disea. 2012 Feb 07;35(5):847–57. doi: 10.1007/s10545-011-9446-x.