Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet)

Inclusion Criteria: * LGDM diagnosed, * Identified gene defect location and gene editing proved feasible, * Age ≥14 years, * Patient in treatment in the department at Charité, Universitätsmedizin Berlin, Muscle Research Unit and Outpatient Clinic for Muscle Disorders, * Signed informed consent Exclusion Criteria: * Acute or chronic inflammatory local or systemic disease * Coagulation disorder * Known complications due to local anesthesia, * Congenital heart defect, cardiac arrhythmia, * Pathology of the airways such as micrognathia * Pierre Robin Sequence * Central hypoventilation syndrome/Ondine syndrome * Significant other medical or psychiatric illness * Positive serology for HIV and/or hepatitis A, B, C * Pregnant or lactating women * Known allergic reaction to constituents of the cryopreservation medium