Towards the development of novel cell therapy options for MSMD patients

About this project

Mendelian susceptibility to mycobacterial disease (MSMD) is a genetic disorder that is caused by mutations in the IFN-γ signaling cascade and predisposes patients to severe infections with low pathogenic mycobacteria like the BCG vaccine. Especially patients with autosomal recessive, complete IFN-γR1 deficiency have poor prognosis and often die in early childhood as a result of disseminated infection with BCG and/or other environmental mycobacteria. In cooperation with Dr. Lachmann, Institute of Experimental Hematology at MHH, we are developing novel iPSC based treatment options for MSMD patients.