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Targeted Nanoparticle Delivery For In Utero Treatment Of Congenital Disorders, Valerie Luks

Yale Medicine Thesis Digital Library

Congenital disorders represent a large unmet clinical need with significant morbidity and premature mortality resulting from mutations and anomalies that can be diagnosed, and eventually treated, in utero. Nanoparticles (NP) have been used as versatile protective vessels for the delivery of a variety of therapeutic agents. Importantly, the potency of NPs can be amplified by modifications to the molecular composition as well as to the delivery to allow targeting to specific tissues. We hypothesize that optimization of NP targeting both by nanoparticle modification and the route of administration can increase overall uptake in specific cells and tissues of interest. To …