Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.

In gastrocnemius muscle cells, inflammatory CD68+ macrophages and CD8+ T cells were reduced in mice treated with INS1201 versus mdx (−) control mice (however, with n = 4 quantified per group, this did not reach statistical significance); inflammatory cells were generally absent in the WT mouse tissue ( Figure S2 ).

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