Dr. Rosa Bacchetta’s team publishes Science Advances paper on CRISPR for IPEX syndrome
Posted on May 13th, 2020
SPARK scholar Dr. Rosa Bacchetta and team featured in SPARK’s blog Bridging the Gap publishes Science Advances paper on CRISPR-based gene correction of the gene that causes rare disease IPEX syndrome. The team showed that a CRISPR-based gene editing platform successfully edited the FOXP3 gene in HPSCs, which maintained differentiation in vitro and in vivo, and in T cells from IPEX patients.