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miRNAs that regulate the IGF-1 signaling are prime targets for the development of novel therapies for several disease including cancer ( Table 1 )

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NPI-001s advantages as a therapy, based on preclinical models, include: Ability to cross blood-brain 33 and blood-retinal barriers 2 Greater cell permeability than NAC 34 Approximately 3-fold more effective than oral NAC 32 , 31 NPI-001 oral tablets have been evaluated in a Phase 1/2 clinical trial (NCT04355689) in patients with RP associated with USH, demonstrating clinical proof of concept

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