Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington's disease phenotypes in R6/2 mice
Danielle A. Simmons, Sridhar Selvaraj, Tingshuo Chen, Gloria Cao, Talita Sauto Camelo, Tyne L.M. McHugh, Selena Gonzalez, Renata M. Martin, Juste Simanauskaite, Nobuko Uchida, Matthew Hebden Porteus, Frank M. Longo
This study shows that human striatal progenitor cells overexpressing BDNF and with inducible safety switches improve HD symptoms in R6/2 mice.
Molecular Therapy — Methods & Clinical Development · Jan 22, 2025Peer-reviewedNeuroscience