Article

Combined RNA interference and gene replacement therapy targeting MFN2 for the treatment of Charcot-Marie-Tooth type 2A

2023-04-26

Abstract excerpt

<title>Abstract</title> <p>Mitofusin-2 (MFN2) is an outer mitochondrial membrane protein essential for mitochondrial networking in most cells. Autosomal dominant mutations in the <italic>MFN2 </italic>gene cause Charcot-Marie-Tooth type 2A disease (CMT2A), a severe and disabling sensory-motor neuropathy that impacts the entire nervous system. Here, we propose a novel therapeutic strategy tailored to correcting th...

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Literature Corpus work: 4242390c-2c54-5294-a28c-e914dfc24bed
DOI: 10.21203/rs.3.rs-2817290/v1

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