Platelet-directed Gene Therapy

Platelets are anuclear blood cells of only 2µm size with an irrecoverable role in hemostasis, but also important functions in inflammation, angiogenesis, tumor growth and innate immunity. Platelets are produced by megakaryocytes in the bone marrow, circulate through the blood stream within the blood vessels and maintain inactive (resting) until activated by environmental signals such as endothelial damage.

Platelets transport numerous proteins within their granules, which can be released after platelet activation, therefore act as "natural carriers". Theses natural characteristic of platelets make these cells an interesting target for cell therapy.

As platelets have no genome, the modification is performed at earlier step of differentiation, the megakaryocyte, hematopoietic progenitor, HSC or even the pluripotent stem cell. For the genetic modification we use lentiviral vectors with transcriptional control that targets expression to megakaryocytes. These vectors can be used to treat inherited platelets disorders. By protein modifications we can load proteins into the secretory platelet granules. Finally, we are interested in the in vitro production of platelets from ES/iPS cells.

PhD students
Simon Pöllmann

Scientific laboratory head
Prof. Ute Modlich