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For interventional trials, we recommend protocolized lot release criteria, recipient risk stratification, and co-primary safety endpoints (e.g., serious infection rate, MDRO colonization), alongside efficacy readouts
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Similar content being viewed by others Log in or create a free account to read this content Gain free access to this article, as well as selected content from this journal and more on nature.com or Abbreviations COX: cytochrome c oxidase CS: citrate synthase CTNS: cystinosin GSH: glutathione GSSG: oxidized glutathione NC: nephropathic cystinosis PG: pyroglutamic acid References Gahl WA, Bashan N, Tietze F, Bernardini I, Schulman JD 1982 Cystine transport is defective in isolated leukocyte lysosomes from patients with cystinosis