S21-023 CRISPR-based FOXP3 gene Engineered T cells and Hematopoietic Stem Cell Precursors to treat IPEX syndrome patients IPEX syndrome is a severe autoimmune disease with limited treatment options caused by mutations in the forkhead box protein 3 (FOXP3) gene, which plays a critical role in immune regulation. Rosa Bacchetta Maria-Grazia Roncarolo Matthew Porteus Marianne Goodwin
S19-517 Three-Recombinase-Dependent Viral Expression for Increased Neuron Specificity Stanford researchers have discovered a method (Triplesect) to express molecular payloads using three-recombinase-dependent viral expression. Karl Deisseroth Charu Ramakrishnan Yoon Seok Kim Lief Fenno