| Title : Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models - Ziolkowska_2025_Sci.Transl.Med_17_eadj1445 |
| Author(s) : Ziolkowska EA , Jansen MJ , Williams LL , Wang SH , Eultgen EM , Takahashi K , Le SQ , Nelvagal HR , Sharma J , Sardiello M , DeBosch BJ , Dickson PI , Anderson JB , Sax SE , Wright CM , Bradley RP , Whiteman IT , Makita T , Grider JR , Sands MS , Heuckeroth RO , Cooper JD |
| Ref : Sci Transl Med , 17 :eadj1445 , 2025 |
| Abstract : |
| PubMedSearch : Ziolkowska_2025_Sci.Transl.Med_17_eadj1445 |
| PubMedID: 39813314 |
| Gene_locus related to this paper: mouse-ppt , human-PPT1 |
| Title : Synergistic effects of treating the spinal cord and brain in CLN1 disease - Shyng_2017_Proc.Natl.Acad.Sci.U.S.A_114_E5920 |
| Author(s) : Shyng C , Nelvagal HR , Dearborn JT , Tyynela J , Schmidt RE , Sands MS , Cooper JD |
| Ref : Proc Natl Acad Sci U S A , 114 :E5920 , 2017 |
| Abstract : |
| PubMedSearch : Shyng_2017_Proc.Natl.Acad.Sci.U.S.A_114_E5920 |
| PubMedID: 28673981 |
| Gene_locus related to this paper: mouse-ppt |
| Title : Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis - Lu_2015_Mol.Genet.Metab_116_98 |
| Author(s) : Lu JY , Nelvagal HR , Wang L , Birnbaum SG , Cooper JD , Hofmann SL |
| Ref : Mol Genet Metab , 116 :98 , 2015 |
| Abstract : |
| PubMedSearch : Lu_2015_Mol.Genet.Metab_116_98 |
| PubMedID: 25982063 |
| Gene_locus related to this paper: mouse-ppt |