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Although the effects of systemically administered hydroxychloroquine have not been evaluated in any AAV gene therapy studies, it is likely that hydroxychloroquine would need to be administered in the weeks prior to AAV delivery to allow hydroxychloroquine to reach appropriate tissue concentrations within the eye

Alterations in sphingolipid composition and mitochondrial bioenergetics represent synergistic therapeutic vulnerabilities linked to multidrug resistance in leukemia

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