Article

Ex vivo long-term expansion of human hematopoietic stem and progenitor cells as a tool for modeling vector integration sites and clonality

2025-11-17

Abstract excerpt

<title>Abstract</title> <p> <bold>Background</bold> : Gene therapy (GT) using retroviral vectors (RVs) is efficacious in treating monogenic diseases. However, there is an inherent risk for severe adverse effects due to insertional mutagenesis. Preclinical safety assessment and patient monitoring are inevitable in GT. To assess the genotoxic risk of novel RV vectors, mainly murine hematopoietic stem and progenit...

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Literature Corpus work: 6287cf8b-29d0-542c-8ace-c720e9f6dfb6
DOI: 10.21203/rs.3.rs-7982018/v1

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