Article

Megabase-Scale Transgene De-Duplication to Generate a Functional Single-Copy Full-Length Human DMD Transgenic Mouse Model

2024-03-26

Abstract excerpt

The development of sequence-specific precision treatments like CRISPR gene-editing therapies for Duchenne Muscular Dystrophy (DMD) requires sequence humanised animal models to enable the direct clinical translation of tested strategies. The current available integrated transgenic mouse model containing the full-length human DMD gene, Tg(DMD)72Thoen/J (hDMDTg), has been found to have two copies of the transgene pe...

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Literature Corpus work: f84b9878-10fe-5fde-92d0-6e69859d1fd0
DOI: 10.1101/2024.03.25.586713

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