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This study evaluates age related effects on CSF delivered lipid-siRNA conjugates, assessing biodistribution, safety, and efficacy. Comparisons between conjugate designs highlights structure dependent performance and provides insight into optimizing siRNA therapeutics for neurodegenerative disease treatment in aged CNS contexts.
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Evaluating the effects of aging on biodistribution and gene silencing activity of lipid-siRNA conjugates delivered into cerebrospinal fluid
Molecular Therapy Family of Journals