Key message
In this study, we were able to show that hematopoietic stem cell and gene therapy can prevent in vivo infections by Mycobacteria spp. using different safety- improved self- inactivating lentiviral vectors in mice with genetic predispositions to mycobacterial infections.
Translational perspective
This study laid the foundation for a new treatment approach for MSMD patients suffering from IFNgR1 deficiency by the use of hematopoietic stem cell and gene therapy. This is of clinical relevance as patients suffering from autosomal recessive, complete IFNgR1 deficiency have poor prognosis and often die in early childhood as a result of disseminated infection with BCG and/or other environmental mycobacteria.