Dr. Lee’s team is testing RNA editing gene therapy for Fragile X, aiming to repair FMR1 RNA and restore missing protein — targeted, reversible, promising. The post Gene Therapeutic Development for Fragile X Syndrome 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 syndrome hippocampal organoids show neuron–glia imbalance. This team will map disrupted gene networks and test PDE inhibitors to restore brain function. The post FMRP Regulatory Role in Human Hippocampal Development and Therapeutic Interventions in Fragile X appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
This project explores the role of oligodendrocytes in Fragile X. The team will test if improving these cells' function can restore normal brain activity to treat Fragile X. The post Oligodendrocytes: a Potential Route to Treat Fragile X Syndrome 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 syndrome researchers are studying how estrogen receptors shape brain activity and may explain why males and females experience symptoms differently. The post Sex Differences and the Role of Estrogen Receptors in Fragile X 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 syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA. The post Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder. The post Validate a Novel Antisense Oligonucleotide Therapeutic for Fragile X Syndrome 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
This grant is funding AI-driven drug discovery, advanced mouse behavior tracking, and gene expression analysis to uncover new treatments for Fragile X syndrome. The post Evaluating Novel Drug Candidates for Fragile X Syndrome Using the Live Mouse Tracker 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 syndrome research explores insulin and cAMP pathway crosstalk, mapping brain metabolism to guide treatment strategies. The post Metabolic and Mitochondrial Crosstalk in Insulin and cAMP Pathways in Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome