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Previously, we reported that the Morc2a p.S87L mouse model exhibited neuropathy and muscular dysfunction through DNA damage accumulation.<\/jats:p>\n                  <jats:p>In the present study, we analysed the gene expression of Morc2a p.S87L mice and designated the primary causing factor. We investigated the pathological pathway using Morc2a p.S87L mouse embryonic fibroblasts and human fibroblasts harbouring MORC2 p.R252W. We subsequently assessed the therapeutic effect of gene therapy administered to Morc2a p.S87L mice.<\/jats:p>\n                  <jats:p>This study revealed that Morc2a p.S87L causes a protein synthesis defect, resulting in the loss of function of Morc2a and high cellular apoptosis induced by high hydroxyl radical levels. We considered the Morc2a GHKL ATPase domain as a therapeutic target because it simultaneously complements hydroxyl radical scavenging and ATPase activity. We used the adeno-associated virus (AAV)-PHP.eB serotype, which has a high CNS transduction efficiency, to express Morc2a or Morc2a GHKL ATPase domain protein in vivo. Notably, AAV gene therapy ameliorated neuropathy and muscular dysfunction with a single treatment. Loss-of-function characteristics due to protein synthesis defects in Morc2a p.S87L were also noted in human MORC2 p.S87L or p.R252W variants, indicating the correlation between mouse and human pathogenesis.<\/jats:p>\n                  <jats:p>In summary, CMT2Z is known as an incurable genetic disorder, but the present study demonstrated its mechanisms and treatments based on established animal models. This study demonstrates that the Morc2a p.S87L variant causes hydroxyl radical-mediated neuropathy, which can be rescued through AAV-based gene therapy.<\/jats:p>","DOI":"10.1093\/brain\/awae017","type":"journal-article","created":{"date-parts":[[2024,1,16]],"date-time":"2024-01-16T14:44:06Z","timestamp":1705416246000},"page":"2114-2127","source":"Crossref","is-referenced-by-count":9,"title":["<i>Morc2a<\/i>\n                    variants cause hydroxyl radical-mediated neuropathy and are rescued by restoring GHKL ATPase"],"prefix":"10.1093","volume":"147","author":[{"given":"Hye Yoon","family":"Chung","sequence":"first","affiliation":[{"name":"Graduate School of International Agricultural Technology and Institute of Green-Bio Science and Technology, Seoul National University , Kangwon 25354 , Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Geon Seong","family":"Lee","sequence":"additional","affiliation":[{"name":"Graduate School of International Agricultural 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