Article

Characterization of a novel zebrafish model of <i>MTMR5</i> -associated CMT4B3

2024-04-20

Abstract excerpt

Biallelic loss of expression/function variants in MTMR5 cause the inherited peripheral neuropathy Charcot-Marie-Tooth (CMT) Type 4B3. There is an incomplete understanding of the disease pathomechanism(s) underlying CMT4B3, and despite its severe clinical presentation, currently no disease modifying therapies. A key barrier to the study of CMT4B3 is the lack of pre-clinical models that recapitulate the clinical an...

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Literature Corpus work: c4f01ca1-b66b-5f0c-9726-956e21a5c084
DOI: 10.1101/2024.04.18.590157

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