Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

Abstract excerpt

A recent clinical trial for adrenoleukodystrophy (ALD) showed the efficacy and safety of lentiviral vector (LV) gene transfer in hematopoietic stem progenitor cells. However, several common insertion sites (CIS) were found in patients' cells, suggesting that LV integrations conferred a selective advantage. We performed high-throughput LV integration site analysis on human hematopoietic stem progenitor cells...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Herpesvirus Infections and Treatments
• Share to RNA Interference and Gene Delivery
• Share to Virus-based gene therapy research