Multifunctional Anti-HIV-1 HSPC Gene Therapy with Negative Selection Marker Shows Feasibility and Safety in Pigtail Macaques
Evaluation of Anti-HIV-1 HSPC-Based Gene Therapy with Negative Selection Marker in Pigtail Macaques.
Abstract
Autologous HSPC gene therapy, delivering anti-HIV-1 payloads via lentiviral vector, achieved safe hematopoietic recovery and sustained multilineage gene marking in pigtail macaques; modified cells expanded upon SHIV challenge.
