Fast, multiplexable and efficient somatic gene deletions in adult mouse skeletal muscle fibers using AAV-CRISPR/Cas9


Thürkauf et al. establish an efficient CRISPR/Cas9 genome editing system for targeted, multiplexable gene knockouts in skeletal muscle fibers of adult mice. The system effectively combines Cre-mediated skeletal muscle fiber-specific Cas9 expression with a myotropic adeno-associated virus-mediated sgRNA delivery. By knocking out key genes for the integrity of the neuromuscular junction and for skeletal muscle fiber growth, they demonstrate the feasibility of local and systemic skeletal muscle fiber-specific gene knockouts over traditional knockout mouse models.


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