UMass Chan Medical School licenses RNA-based Fragile X treatment approach using ASOs to QurAlis, moving gene-targeted therapy closer to clinical trials. The post UMass Chan Medical School Licenses RNA-Based Therapy to QurAlis, Advancing a New Era in Fragile X Treatment appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Fragile X research is at a turning point. Two late-stage clinical trials, zatolmilast (EXPERIENCE) and ZYN002 (RECONNECT), could soon deliver the first approved treatments. FRAXA is also funding innovative approaches like ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X. The post Fragile X Research Update: A Turning Point for Treatments and Curative Approaches appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
A new FRAXA grant funds UMass Chan researchers using ASOs in neurons and organoids to correct FMR1 mis-splicing and restore critical FMRP protein. The post ASO Rescue of FMR1 Mis-Splicing in Neurons and Mitigation of Fragile X Deficits appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome