Patient-derived muscle stem cells for the treatment of muscle wasting and muscular defects

Institution: Charité Campus Buch, Experimental and Clinical Research Center
Applicant: Prof. Dr. Simone Spuler, Charité Universitätsmedizin Berlin und Max-Delbrück Zentrum für Molekulare Medizin
Funding line:
Translational Research
Project plan to obtain, propagate, and transplant  primary human muscle stem in an autologous setting.

Project partner: Prof. Dr. Anne-Karoline Ebert, Leiterin, Pädiatrische Urologie, Universitätsklinikum Ulm

Skeletal-muscle stem cells repair muscle. We want to utilize this potential for the therapy of muscle wasting and muscle defects. Therefore, we developed muscle stem cells as an advanced therapeutic medicinal product (ATMP) to be transplanted in an autologous manner (PHSats).

Verena Schöwel, Janine Kieshauer, Simone Spuler and Andreas Marg (from left to right) are developing a cell therapy using primary human muscle stem cells.

We isolate, multiply, and store satellite cells in a process that was developed by us. The regenerative potential of PHSats was demonstrated in preclinical experiments, and safety issues were addressed. We are currently preparing the first-in-man clinical trial for repairing a defective urethral sphincter in a developmental disorder.

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