Quantifying the Mutational Landscape of Retroviral and Lentiviral Vectors in Gene Therapy Patients
This study investigates mutational signatures within the integrated transgene of patients treated with retroviral and lentiviral ADA-SCID gene therapy. DNA sequencing of ADA-SCID patient cells treated with a lentiviral vector revealed a high frequency of guanine-to-adenosine base substitutions across the integrated transgene, driven by APOBEC3 cytosine deaminases. DNA sequencing datasets are provided here of all patient sequencing for variant analysis, mutation selection over time, and APOBEC3-mediated mutagenesis occurrence.