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
Explore Turner Lab's novel approach to developing definitive treatment for Fragile X syndrome with a Tat-conjugated, truncated FMRP protein. This innovative strategy aims to restore brain circuit function and reduce abnormal behaviors linked to Fragile X by directly addressing the cause of Fragile X: a missing protein. The post A Tat-Conjugate Approach 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
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