| 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 |
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Ref :
Sci Transl Med , 17 :eadj1445 , 2025 |
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Abstract :
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| PubMedSearch : Ziolkowska_2025_Sci.Transl.Med_17_eadj1445 |
| PubMedID: 39813314 |
| Gene_locus related to this paper:
mouse-ppt , human-PPT1 |