Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington’s disease phenotypes by Danielle A. Simmons, Sridhar Selvaraj, Tingshuo Chen, Gloria Cao, Talita Souto Camelo, Tyne L.M. McHugh, Selena Gonzalez, Renata M. Martin, Juste Simanauskaite, Nobuko Uchida, Matthew H. Porteus, Frank M. Longo

Get full text

Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells by Simon N. Chu, Eric Soupene, Devesh Sharma, Roshani Sinha, Travis McCreary, Britney Hernandez, Huifeng Shen, Beeke Wienert, Chance Bowman, Han Yin, Benjamin J. Lesch, Kun Jia, Kathleen A. Romero, Zachary Kostamo, Yankai Zhang, Tammy Tran, Marco Cordero, Shota Homma, Jessica P. Hampton, James M. Gardner, Bruce R. Conklin, Tippi C. MacKenzie, Vivien A. Sheehan, Matthew H. Porteus, M. Kyle Cromer

Get full text