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