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Gene therapy strategies that aim to increase expression of a functional dystrophin protein (mini-dystrophin) are under investigation. The ability to accurately quantify dystrophin\/mini-dystrophin is essential in assessing the level of gene transduction. We demonstrated the validation and application of a novel peptide immunoaffinity liquid chromatography\u2013tandem mass spectrometry (IA-LC-MS\/MS) assay. Data showed that dystrophin expression in Becker muscular dystrophy and DMD tissues, normalized against the mean of non-dystrophic control tissues (<jats:italic>n<\/jats:italic>\u2009=\u200920), was 4\u201384.5% (mean 32%, <jats:italic>n<\/jats:italic>\u2009=\u200920) and 0.4\u201324.1% (mean 5%, <jats:italic>n<\/jats:italic>\u2009=\u200920), respectively. In a DMD rat model, biceps femoris tissue from dystrophin-deficient rats treated with AAV9.hCK.Hopti-Dys3978.spA, an adeno-associated virus vector containing a mini-dystrophin transgene, showed a dose-dependent increase in mini-dystrophin expression at 6 months post-dose, exceeding wildtype dystrophin levels at high doses. Validation data showed that inter- and intra-assay precision were \u226420% (\u226425% at the lower limit of quantification [LLOQ]) and inter- and intra-run relative error was within \u00b120% (\u00b125% at LLOQ). IA-LC-MS\/MS accurately quantifies dystrophin\/mini-dystrophin in human and preclinical species with sufficient sensitivity for immediate application in preclinical\/clinical trials.<\/jats:p>","DOI":"10.1038\/s41434-021-00300-7","type":"journal-article","created":{"date-parts":[[2021,11,5]],"date-time":"2021-11-05T02:02:54Z","timestamp":1636077774000},"page":"608-615","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":30,"title":["Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy"],"prefix":"10.1038","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8820-8919","authenticated-orcid":false,"given":"Vahid","family":"Farrokhi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7229-9131","authenticated-orcid":false,"given":"Jason","family":"Walsh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joe","family":"Palandra","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5261-3381","authenticated-orcid":false,"given":"Joanne","family":"Brodfuehrer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3582-3600","authenticated-orcid":false,"given":"Teresa","family":"Caiazzo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane","family":"Owens","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Binks","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8768-414X","authenticated-orcid":false,"given":"Srividya","family":"Neelakantan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7829-6927","authenticated-orcid":false,"given":"Florence","family":"Yong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pinky","family":"Dua","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Le Guiner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8476-5427","authenticated-orcid":false,"given":"Hendrik","family":"Neubert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,11,5]]},"reference":[{"key":"300_CR1","unstructured":"Muscular Dystrophy Association. 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