WP7 will exploit the newly developed technologies to treat dominant muscle genetic disorders and muscle ageing and senescence. A pathogenic CTG repeat in the human DM1 gene of transgenic mice will be deleted in skeletal muscle, heart, and diaphragm by CRISPR/Cas9 delivery. Targeted epigenetic editing will be used to reduce expression or activate transcription of key genes in order to attenuate and/or prevent sarcopenia and cardiac dysfunction in aging mice.
Specific WP’s objectives:
- To test efficacy of innovative skeletal and cardiac muscle-specific gene therapy platform based on CRISPR/Cas9 gene editing to cure dominant genetic disorders (DM1)
- To test efficacy and safety of targeted epigenetic editing approaches in preventing senescent cells in aged skeletal and cardiac muscles
- To test efficacy and safety of targeted epigenetic approaches in inducing autophagy in aged skeletal and cardiac muscles
Tasks:
- Task 7.1: Preclinical validation of gene editing in DM1 mice
- Task 7.2: Prevention of senescence entry through epigenetic editing to prevent muscle sarcopenia and cardiac dysfunction in aging mice
- Task 7.3: Induction of autophagy through epigenetic editing to attenuate sarcopenia and cardiac dysfunction in aging mice
Timeline:
WP leader: Pura Muñoz (UPF)
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