{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T19:56:23Z","timestamp":1793044583433,"version":"4.5.0"},"reference-count":75,"publisher":"MDPI AG","issue":"12","license":[{"start":{"date-parts":[[2021,12,15]],"date-time":"2021-12-15T00:00:00Z","timestamp":1639526400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000050","name":"National Heart Lung and Blood Institute","doi-asserted-by":"publisher","award":["R01 HL149452"],"award-info":[{"award-number":["R01 HL149452"]}],"id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01 HL140468"],"award-info":[{"award-number":["R01 HL140468"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceutics"],"abstract":"<jats:p>Gene therapy is a good alternative for determined congenital disorders; however, there are numerous limitations for gene delivery in vivo including targeted cellular uptake, intracellular trafficking, and transport through the nuclear membrane. Here, a modified G5 polyamidoamine (G5 PAMAM) dendrimer\u2013DNA complex was developed, which will allow cell-specific targeting to skeletal muscle cells and transport the DNA through the intracellular machinery and the nuclear membrane. The G5 PAMAM nanocarrier was modified with a skeletal muscle-targeting peptide (SMTP), a DLC8-binding peptide (DBP) for intracellular transport, and a nuclear localization signaling peptide (NLS) for nuclear uptake, and polyplexed with plasmid DNA containing the GFP-tagged microdystrophin (\u00b5Dys) gene. The delivery of \u00b5Dys has been considered as a therapeutic modality for patients suffering from a debilitating Duchenne muscular dystrophy (DMD) disorder. The nanocarrier\u2013peptide\u2013DNA polyplexes were prepared with different charge ratios and characterized for stability, size, surface charge, and cytotoxicity. Using the optimized nanocarrier polyplexes, the transfection efficiency in vitro was determined by demonstrating the expression of the GFP and the \u00b5Dys protein using fluorescence and Western blotting studies, respectively. Protein expression in vivo was determined by injecting an optimal nanocarrier polyplex formulation to Duchenne model mice, mdx4Cv. Ultimately, these nanocarrier polyplexes will allow targeted delivery of the microdystrophin gene to skeletal muscle cells and result in improved muscle function in Duchenne muscular dystrophy patients.<\/jats:p>","DOI":"10.3390\/pharmaceutics13122159","type":"journal-article","created":{"date-parts":[[2021,12,15]],"date-time":"2021-12-15T21:47:36Z","timestamp":1639604856000},"page":"2159","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery"],"prefix":"10.3390","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0875-4248","authenticated-orcid":false,"given":"Jessica","family":"Hersh","sequence":"first","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"The Dr. John T. McDonald Foundation Bionanotechnology Institute, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jos\u00e9 Manuel","family":"Condor Capcha","sequence":"additional","affiliation":[{"name":"Interdisciplinary Stem Cell Institute and Division of Cardiology, Department of Medicine, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camila","family":"Iansen Irion","sequence":"additional","affiliation":[{"name":"Interdisciplinary Stem Cell Institute and Division of Cardiology, Department of Medicine, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guerline","family":"Lambert","sequence":"additional","affiliation":[{"name":"Interdisciplinary Stem Cell Institute and Division of Cardiology, Department of Medicine, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mauricio","family":"Noguera","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"The Dr. John T. McDonald Foundation Bionanotechnology Institute, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohit","family":"Singh","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"The Dr. John T. McDonald Foundation Bionanotechnology Institute, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Avinash","family":"Kaur","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8212-1791","authenticated-orcid":false,"given":"Emre","family":"Dikici","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"The Dr. John T. McDonald Foundation Bionanotechnology Institute, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joaqu\u00edn J.","family":"Jim\u00e9nez","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"Dr. Phillip Frost Department of Dermatology and Cutaneous Surgery, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lina A.","family":"Shehadeh","sequence":"additional","affiliation":[{"name":"Interdisciplinary Stem Cell Institute and Division of Cardiology, Department of Medicine, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"Daunert","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"The Dr. John T. McDonald Foundation Bionanotechnology Institute, University of Miami, Miami, FL 33136, USA"},{"name":"Clinical and Translational Science Institute, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sapna K.","family":"Deo","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, Leonard M. Miller School of Medicine, University of Miami, Miami, FL 33136, USA"},{"name":"The Dr. John T. McDonald Foundation Bionanotechnology Institute, University of Miami, Miami, FL 33136, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2021,12,15]]},"reference":[{"key":"ref_1","unstructured":"Genetic Alliance, and District of Columbia Department of Health (2010). Appendix G Single-Gene Disorders. Understanding Genetics: A District of Columbia Guide for Patients and Health Professionals, Genetic Alliance."},{"key":"ref_2","first-page":"192","article-title":"Rare Genetic Disorders: Learning About Genetic Disease Through Gene Mapping, SNPs, and Microarray Data","volume":"1","author":"Chial","year":"2008","journal-title":"Nat. Educ."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1016\/0887-8994(95)00251-0","article-title":"Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy","volume":"14","author":"Boland","year":"1996","journal-title":"Pediatr. 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