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8f), which suggests that nucleotide salvage might instead be linked to our observed mitochondrial phenotypes

It is precisely for this reason that quality documentation is so important: in the absence of the regulatory framework for a medicinal product, the certificate of analysis and traceability are the only reliable indicators of the actual contents of the vial

This was not the case, as recently described in a publication in Gastro Hep Advances , but the study opened our eyes to bile acid diarrhea as a debilitating and underdiagnosed condition with no effective therapies

Phase 1 studies involving ex vivoedited T cells (e.g., TCR knockout to reduce graft rejection) using lentiviral vectors are ongoing, but definitive efficacy data have not yet been published [139]