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Gene therapy for ATP1A3-Related Neurologic Disorders
Unmet Need Children with Alternating Hemiplegia of Childhood can have episodes of temporary paralysis, dystonia, and severe behavioral problems. The prevalence of AHC is estimated to be 1 in one million to 1 out of…
A tool to facilitate high affinity binding between beta-Arrestin and GPCR
Unmet Need Beta-arrestins are a highly conserved family of cytosolic adaptor proteins that contribute to a multitude of physiological functions through a canonical interaction with various G protein-coupled receptors (GPCRs). Through agonist binding to GPCRs,…