Innovative gene therapy concepts for Mendelian Susceptibility to Mycobacterial Disease (MSMD)

Project start
Institution: Medizinische Hochschule Hannover, Institut für Experimentelle Hämatologie
Applicant: PD Dr. Nico Lachmann
EKFS funding line: First and Second Applications
: PD Dr. Nico Lachmann (left) is working together with Patrick Blank (middle) and Dr. Miriam Hetzel (right) on the new gene therapy approach

The human immune system is equipped with a variety of different blood cells to fight a multitude of pathogens each day. Mutations in the genome can cause phagocytes like macrophages to become non-functional and these cells then fail to clear pathogens like mycobacteria. This is the case for the disease MSMD, in which patients suffer from severe and sometimes lethal mycobacterial infections. The funded project tries to develop novel therapy options for these patients. We investigate whether genetic correction of hematopoietic stem cells and the subsequent transplantation of these cells can save a relevant mouse model from otherwise lethal mycobacterial infections.