Article

Gene therapy with hematopoietic stem and progenitor cell for monogenic disorders: a systematic review and meta-analysis

2021-06-12

Abstract excerpt

<title>Abstract</title> <p>To provide an assessment of the safety of ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPC), we reviewed in a systematic manner the literature on monogenic diseases to describe survival, genotoxicity and engraftment of gene corrected HSPC, across vector platforms and diseases. From 1995 to 2020, 55 trials for 14 diseases met inclusion criteria and 406 patient...

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Literature Corpus work: f418181d-02b3-5942-9ee4-3408f974cfab
DOI: 10.21203/rs.3.rs-596185/v1

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