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In cases such as Friedreich's ataxia (FRDA), which is characterized by mitochondrial dysfunction, drug conjugated NPs can be combined with gene therapy for enhancing the delivery of genetic material to the target cells

In the original mouse work it was described as well-tolerated, but that is the limit of the in-vivo safety evidence, and it is mouse-only

Discontinuation due to GI intolerance was 2.9% versus 8.4%