Bowel dysmotility and enteric neuron degeneration in lysosomal storage disease mice is prevented by gene therapy
Matthew J. Jansen, Letitia L. Williams, Sophie H. Wang, Elizabeth M. Eultgen, Keigo Takahashi, Hemanth R. Nelvagal, Jaiprakash Sharma, Marco Sardiello, Brian J. DeBosch, Jessica B. Anderson, Sophie E. Sax, Christina M. Wright, Takako Makita, John R. Grider, Mark S. Sands, , Jonathan D. Cooper
This paper shows gene therapy prevents bowel motility issues and enteric neuron loss in mouse models of lysosomal storage diseases.
bioRxiv · May 26, 2023 · PreprintNeuroscience