Article

Development of evolutionarily conserved viral integration sites as safe harbors for human gene therapy

2023-09-10

Abstract excerpt

Gene transfer into CD34 + hematopoietic stem and progenitor cells (HSPCs) involving integrating viral vectors has unpredictable outcomes including potential adverse events like leukemogenesis, resulting from insertional mutagenesis. Therefore, identifying and characterizing genome safe harbor (GSH) sites where exogenous gene material can be safely integrated into adult progenitor and stem cells is critically impo...

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Literature Corpus work: be751af2-4ce5-5a00-98cc-35ba9ff044b2
DOI: 10.1101/2023.09.08.556857

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