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with Prof. Dr. med. Simone Spuler

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Muscular dystrophies comprise of about fifty different monogenic diseases with severe, debilitating clinical courses and without available therapies. The overall incidence is 1 : 2.500. Skeletal muscle is ubiquitous in the body and not transplantable as an entire organ. Combined cell and gene therapy would be a perfect approach to treat muscular dystrophies, but their success was hampered by the lack of 1. potent muscle stem cells ready for clinical use and 2. highly efficient gene correction strategies. In this talk you will hear about how muscle stem cells are returning to the focus of attention in the treatment of muscle wasting. In addition, gene correction strategies derived from the CRISPR/Cas9 techniques are demonstrated in unprecedented efficacy in primary human muscle stem cells.

Prof. Dr. med. Simone Spuler is Head of the Out-Patient Clinic for Muscle Disorders and Senior Group Leader of the research group Myology at the Experimental and Clinical Research Center Charité (ECRC) of the Charité – Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine (MDC). Spuler studied medicine at the University of Würzburg. As a postdoc at UC San Diego, CA (USA) and during a joint appointment between the Max Planck Institute for Neurobiology (Martinsried) and the Dept. of Neurology at LMU Munich, she focused on translational aspects of basic research and clinical application early on. After her residency in general neurology, Spuler did a two-year clinical fellowship in muscle sciences with Andrew G. Engel at the Mayo Clinic in Rochester, MN (USA). Her main research interest is cell- and gene therapy for muscular dystrophies. She initiated a first-in-human clinical trial using muscle stem cells (PHSats) starting May 2021.


The Lecture will be held online via GoToMeeting. To participate in the Lecture, please register here. The registration will be open until maximal participant capacity is reached.

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Friday, 23 April 2021
12:00 – 1:00 pm


via GoToMeeting
A login-link or dial-in number for the phone as well as an access code for the BIH Lecture will be provided the evening before the event.


Please register here.
The registration will be open until maximal participant capacity is reached.