NEW THERAPEUTIC APPROACHES AND THEIR READOUT

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Targeting disease modifier genes is an attractive therapeutic strategy in cases where causative genes are large and mutations are heterogeneous. Here, we report a mutation-independent strategy to upregulate expression of a compensatory disease-modifying gene in congenital muscular dystrophy type 1A (MDC1A) using a CRISPR/dCas9-based transcriptional activation system. MDC1A is caused by nonfunctional laminin α2, which compromises muscle fibers stability and axon myelination in peripheral nerves. Transgenic overexpression of Lama1, encoding a structurally similar Laminin α1, ameliorates muscle wasting and paralysis in MDC1A mouse models, demonstrating its role as a protective disease modifier.

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