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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,…
Enhancing of homing and/or engraftment of cord blood mononuclear cells in the central nervous system
Value Proposition Leukodystrophies are inherited disorders resulting in decreased motor function, muscle rigidity, degeneration of sight and hearing, and are ultimately fatal. There is a great lack of treatment, although cord blood mononuclear (CBM) and…