Title : Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis - Roberts_2012_J.Inherit.Metab.Dis_35_847 |
Author(s) : Roberts MS , Macauley SL , Wong AM , Yilmas D , Hohm S , Cooper JD , Sands MS |
Ref : J Inherit Metab Dis , 35 :847 , 2012 |
Abstract : |
PubMedSearch : Roberts_2012_J.Inherit.Metab.Dis_35_847 |
PubMedID: 22310926 |
Gene_locus related to this paper: mouse-ppt |