Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models.
Ziółkowska 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.
Ziółkowska EA, et al. Among authors: anderson jb.
Sci Transl Med. 2025 Jan 15;17(781):eadj1445. doi: 10.1126/scitranslmed.adj1445. Epub 2025 Jan 15.
Sci Transl Med. 2025.
PMID: 39813314