{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T07:06:28Z","timestamp":1779087988924,"version":"3.51.4"},"reference-count":9,"publisher":"Springer Science and Business Media LLC","issue":"4","license":[{"start":{"date-parts":[[2014,3,12]],"date-time":"2014-03-12T00:00:00Z","timestamp":1394582400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2014,3,12]],"date-time":"2014-03-12T00:00:00Z","timestamp":1394582400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["EMBO Mol Med"],"published-print":{"date-parts":[[2014,4,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>When mitochondrial diseases result from mutations found in the mitochondrial DNA, engineered mitochondrial\u2010targeted nucleases such as mitochondrial\u2010targeted zinc finger nucleases are shown to specifically eliminate the mutated molecules, leaving the wild\u2010type mitochondrial DNA intact to replicate and restore normal copy number. In this issue, Gammage and colleagues successfully apply this improved technology on patients' cells with two types of genetic alterations responsible for neuropathy ataxia and retinitis pigmentosa (NARP) syndrome and Kearns Sayre syndrome and progressive external ophthalmoplegia (PEO).<\/jats:p>","DOI":"10.1002\/emmm.201303769","type":"journal-article","created":{"date-parts":[[2014,3,19]],"date-time":"2014-03-19T14:38:30Z","timestamp":1395239910000},"page":"434-435","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":21,"title":["A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells"],"prefix":"10.1038","volume":"6","author":[{"given":"Carlos T","family":"Moraes","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2014,3,12]]},"reference":[{"key":"BFEMMM201303769_CR1","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1038\/gt.2011.196","volume":"19","author":"SR Bacman","year":"2012","unstructured":"Bacman SR, Williams SL, Duan D, Moraes CT (2012) Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9\u2010mediated delivery of a mitochondria\u2010targeted restriction endonuclease. Gene Ther 19: 1101\u20131106","journal-title":"Gene Ther"},{"key":"BFEMMM201303769_CR2","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1038\/gt.2010.25","volume":"17","author":"SR Bacman","year":"2010","unstructured":"Bacman SR, Williams SL, Garcia S, Moraes CT (2010) Organ\u2010specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria\u2010targeted restriction endonuclease. Gene Ther 17: 713\u2013720","journal-title":"Gene Ther"},{"key":"BFEMMM201303769_CR3","doi-asserted-by":"crossref","first-page":"1111","DOI":"10.1038\/nm.3261","volume":"19","author":"SR Bacman","year":"2013","unstructured":"Bacman SR, Williams SL, Pinto M, Peralta S, Moraes CT (2013) Specific elimination of mutant mitochondrial genomes in patient\u2010derived cells by mitoTALENs. Nat Med 19: 1111\u20131113","journal-title":"Nat Med"},{"key":"BFEMMM201303769_CR5","doi-asserted-by":"crossref","first-page":"686","DOI":"10.1016\/j.mito.2011.05.004","volume":"11","author":"PJ Carling","year":"2011","unstructured":"Carling PJ, Cree LM, Chinnery PF (2011) The implications of mitochondrial DNA copy number regulation during embryogenesis. Mitochondrion 11: 686\u2013692","journal-title":"Mitochondrion"},{"key":"BFEMMM201303769_CR6","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1002\/emmm.201303672","volume":"6","author":"PA Gammage","year":"2014","unstructured":"Gammage PA, Rorbacha J, Vincentb AI, Rebarb EJ, Minczuk M (2014) Mitochondrially\u2010targeted ZFNs for selective degradation of pathogenic mitochondrial genomes bearing large\u2010scale deletions or point mutations. EMBO Mol Med 6: 458\u2013466","journal-title":"EMBO Mol Med"},{"key":"BFEMMM201303769_CR4","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/nrg2988","volume":"12","author":"F Mingozzi","year":"2011","unstructured":"Mingozzi F, High KA (2011) Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 12: 341\u2013355","journal-title":"Nat Rev Genet"},{"key":"BFEMMM201303769_CR7","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1038\/gt.2012.2","volume":"20","author":"NJ Palpant","year":"2013","unstructured":"Palpant NJ, Dudzinski D (2013) Zinc finger nucleases: looking toward translation. Gene Ther 20: 121\u2013127","journal-title":"Gene Ther"},{"key":"BFEMMM201303769_CR8","doi-asserted-by":"crossref","first-page":"878","DOI":"10.1038\/nrg3275","volume":"13","author":"EA Schon","year":"2012","unstructured":"Schon EA, DiMauro S, Hirano M (2012) Human mitochondrial DNA: roles of inherited and somatic mutations. Nat Rev Genet 13: 878\u2013890","journal-title":"Nat Rev Genet"},{"key":"BFEMMM201303769_CR9","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1093\/hmg\/3.1.13","volume":"3","author":"M Sciacco","year":"1994","unstructured":"Sciacco M, Bonilla E, Schon EA, DiMauro S, Moraes CT (1994) Distribution of wild\u2010type and common deletion forms of mtDNA in normal and respiration\u2010deficient muscle fibers from patients with mitochondrial myopathy. Hum Mol Genet 3: 13\u201319","journal-title":"Hum Mol Genet"}],"container-title":["EMBO Molecular Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1002\/emmm.201303769.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/emmm.201303769","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1002\/emmm.201303769\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.embopress.org\/doi\/pdf\/10.1002\/emmm.201303769","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1002\/emmm.201303769.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T06:08:51Z","timestamp":1779084531000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1002\/emmm.201303769"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,3,12]]},"references-count":9,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2014,4,1]]}},"alternative-id":["BFEMMM201303769"],"URL":"https:\/\/doi.org\/10.1002\/emmm.201303769","relation":{},"ISSN":["1757-4676","1757-4684"],"issn-type":[{"value":"1757-4676","type":"print"},{"value":"1757-4684","type":"electronic"}],"subject":[],"published":{"date-parts":[[2014,3,12]]},"assertion":[{"value":"12 March 2014","order":1,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The author declares that he has no conflict of interest.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}