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Size, surface charge, and shape determine therapeutic effects of nanoparticles on brain and retinal diseases

A 17-year-old female with a history of epilepsy on carbamazepine, zonisamide, and levetiracetam is hospitalized for breakthrough seizures

A second-generation AAV9 vector with a modified 3UTR and a panel of miRNA binding sites was developed with the goal of biasing transgene expression away from the liver (Sinnett et al

Its role in the control of ketogenesis
