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These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials.", "publicationTitle": "Advanced Drug Delivery Reviews", "publisher": "", "place": "", "date": "June 29, 2015", "volume": "87", "issue": "", "section": "", "partNumber": "", "partTitle": "", "pages": "104-107", "series": "Recent Developments in Oligonucleotide Based Therapeutics", "seriesTitle": "", "seriesText": "", "journalAbbreviation": "Advanced Drug Delivery Reviews", "DOI": "10.1016/j.addr.2015.05.008", "citationKey": "", "url": "http://www.sciencedirect.com/science/article/pii/S0169409X15001015", "accessDate": "2015-11-29T08:39:32Z", "PMID": "", "PMCID": "", "ISSN": "0169-409X", "archive": "", "archiveLocation": "", "shortTitle": "", "language": "", "libraryCatalog": "ScienceDirect", "callNumber": "", "rights": "", "extra": "", "tags": [ { "tag": "Clinical trials", "type": 1 }, { "tag": "Drisapersen", "type": 1 }, { "tag": "Eteplirsen", "type": 1 }, { "tag": "RNA splicing", "type": 1 }, { "tag": "RNA therapeutics", "type": 1 }, { "tag": "exon skipping", "type": 1 } ], "collections": [], "relations": {}, "dateAdded": "2015-11-29T08:39:32Z", "dateModified": "2015-11-29T08:39:32Z" } }, { "key": "EHHWFJEQ", "version": 42, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/EHHWFJEQ", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/EHHWFJEQ", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/NZ62UIIT", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2612502, "username": "aborenstein15", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/aborenstein15", "type": "text/html" } } } }, "data": { "key": "EHHWFJEQ", "version": 42, "parentItem": "NZ62UIIT", "itemType": "attachment", "linkMode": "imported_url", "title": "Snapshot", "accessDate": "2015-11-29T06:39:37Z", "url": "http://www.nejm.org.ccl.idm.oclc.org/doi/full/10.1056/NEJM198906153202405", "note": "", "contentType": "text/html", "charset": "utf-8", "filename": "NEJM198906153202405.html", "md5": "5b3a67cf69c61f4b67f4edcdc6cb5540", "mtime": 1448779177000, "tags": [], "relations": {}, "dateAdded": "2015-11-29T06:39:37Z", "dateModified": "2015-11-29T06:39:37Z" } }, { "key": "NZ62UIIT", "version": 41, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/NZ62UIIT", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/NZ62UIIT", "type": "text/html" } }, "meta": { "createdByUser": { "id": 2612502, "username": "aborenstein15", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/aborenstein15", "type": "text/html" } } }, "creatorSummary": "Mendell et al.", "parsedDate": "1989-06-15", "numChildren": 1 }, "data": { "key": "NZ62UIIT", "version": 41, "itemType": "journalArticle", "title": "Randomized, Double-Blind Six-Month Trial of Prednisone in Duchenne's Muscular Dystrophy", "creators": [ { "creatorType": "author", "firstName": "J.R.", "lastName": "Mendell" }, { "creatorType": "author", "firstName": "R.T.", "lastName": "Moxley" }, { "creatorType": "author", "firstName": "R.C.", "lastName": "Griggs" }, { "creatorType": "author", "firstName": "M.H.", "lastName": "Brooke" }, { "creatorType": "author", "firstName": "G.M.", "lastName": "Fenichel" }, { "creatorType": "author", "firstName": "J.P.", "lastName": "Miller" }, { "creatorType": "author", "firstName": "W.", "lastName": "King" }, { "creatorType": "author", "firstName": "L.", "lastName": "Signore" }, { "creatorType": "author", "firstName": "S.", "lastName": "Pandya" }, { "creatorType": "author", "firstName": "J.", "lastName": "Florence" }, { "creatorType": "author", "firstName": "J.", "lastName": "Schierbecker" }, { "creatorType": "author", "firstName": "J.", "lastName": "Robison" }, { "creatorType": "author", "firstName": "K.", "lastName": "Kaiser" }, { "creatorType": "author", "firstName": "S.", "lastName": "Mandel" }, { "creatorType": "author", "firstName": "C.", "lastName": "Arfken" }, { "creatorType": "author", "firstName": "B.", "lastName": "Gilder" } ], "abstractNote": "DUCHENNE'S muscular dystrophy is an inexorably progressive disease that results in severe disability by the age of 10 to 12 years and in death in early adulthood. Despite recent major advances in the understanding of the molecular genetic defect,1 2 3 4 5 6 no treatment improves or even slows the disorder. We recently observed an increase in muscle strength after six months of prednisone treatment (1.5 mg per kilogram of body weight per day) in an open therapeutic trial in 33 patients with Duchenne's muscular dystrophy.7 That study, which used historical controls,8 suggested that patients with this disorder received a short-term benefit. However, that . . .", "publicationTitle": "New England Journal of Medicine", "publisher": "", "place": "", "date": "June 15, 1989", "volume": "320", "issue": "24", "section": "", "partNumber": "", "partTitle": "", "pages": "1592-1597", "series": "", "seriesTitle": "", "seriesText": "", "journalAbbreviation": "", "DOI": "10.1056/NEJM198906153202405", "citationKey": "", "url": "http://dx.doi.org/10.1056/NEJM198906153202405", "accessDate": "2015-11-29T06:39:30Z", "PMID": "", "PMCID": "", "ISSN": "0028-4793", "archive": "", "archiveLocation": "", "shortTitle": "", "language": "", "libraryCatalog": "Taylor and Francis+NEJM", "callNumber": "", "rights": "", "extra": "PMID: 2657428", "tags": [], "collections": [ "IT64SMGH" ], "relations": {}, "dateAdded": "2015-11-29T06:39:30Z", "dateModified": "2015-11-29T06:39:30Z" } }, { "key": "MNEWN692", "version": 39, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/MNEWN692", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/MNEWN692", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/3927AP57", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2612502, "username": "aborenstein15", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/aborenstein15", "type": "text/html" } } } }, "data": { "key": "MNEWN692", "version": 39, "parentItem": "3927AP57", "itemType": "attachment", "linkMode": "imported_url", "title": "Snapshot", "accessDate": "2015-11-29T06:35:18Z", "url": "http://onlinelibrary.wiley.com.ccl.idm.oclc.org/doi/10.1002/humu.20918/abstract", "note": "", "contentType": "text/html", "charset": "utf-8", "filename": "abstract.html", "md5": "672d4c021cb745896e2f75ad7ce655d7", "mtime": 1448778918000, "tags": [], "relations": {}, "dateAdded": "2015-11-29T06:35:18Z", "dateModified": "2015-11-29T06:35:18Z" } }, { "key": "4HS8Z6ZP", "version": 39, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/4HS8Z6ZP", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/4HS8Z6ZP", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/3927AP57", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2612502, "username": "aborenstein15", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/aborenstein15", "type": "text/html" } } }, "numChildren": 0 }, "data": { "key": "4HS8Z6ZP", "version": 39, "parentItem": "3927AP57", "itemType": "attachment", "linkMode": "imported_url", "title": "Full Text PDF", "accessDate": "2015-11-29T06:35:13Z", "url": "http://onlinelibrary.wiley.com.ccl.idm.oclc.org/doi/10.1002/humu.20918/pdf", "note": "", "contentType": "application/pdf", "charset": "", "filename": "Aartsma-Rus et al. - 2009 - Theoretic applicability of antisense-mediated exon.pdf", "md5": "9828fc8a7488c677388db3489fa32367", "mtime": 1448778915000, "tags": [], "relations": {}, "dateAdded": "2015-11-29T06:35:13Z", "dateModified": "2015-11-29T06:35:15Z" } }, { "key": "3927AP57", "version": 40, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/3927AP57", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/3927AP57", "type": "text/html" } }, "meta": { "createdByUser": { "id": 2612502, "username": "aborenstein15", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/aborenstein15", "type": "text/html" } } }, "creatorSummary": "Aartsma-Rus et al.", "parsedDate": "2009-03-01", "numChildren": 2 }, "data": { "key": "3927AP57", "version": 40, "itemType": "journalArticle", "title": "Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations", "creators": [ { "creatorType": "author", "firstName": "Annemieke", "lastName": "Aartsma-Rus" }, { "creatorType": "author", "firstName": "Ivo", "lastName": "Fokkema" }, { "creatorType": "author", "firstName": "Jan", "lastName": "Verschuuren" }, { "creatorType": "author", "firstName": "Ieke", "lastName": "Ginjaar" }, { "creatorType": "author", "firstName": "Judith", "lastName": "van Deutekom" }, { "creatorType": "author", "firstName": "Gert-Jan", "lastName": "van Ommen" }, { "creatorType": "author", "firstName": "Johan T.", "lastName": "den Dunnen" } ], "abstractNote": "Antisense-mediated exon skipping aiming for reading frame restoration is currently a promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach is mutation specific, but as the majority of DMD patients have deletions that cluster in hotspot regions, the skipping of a small number of exons is applicable to relatively large numbers of patients. To assess the actual applicability of the exon skipping approach, we here determined for deletions, duplications and point mutations reported in the Leiden DMD mutation database, which exon(s) should be skipped to restore the open reading frame. In theory, single and double exon skipping would be applicable to 79% of deletions, 91% of small mutations, and 73% of duplications, amounting to 83% of all DMD mutations. Exon 51 skipping, which is being tested in clinical trials, would be applicable to the largest group (13%) of all DMD patients. Further research is needed to determine the functionality of different in-frame dystrophins and a number of hurdles has to be overcome before this approach can be applied clinically. Hum Mutat 0, 1–7, 2009. © 2009 Wiley-Liss, Inc.", "publicationTitle": "Human Mutation", "publisher": "", "place": "", "date": "March 1, 2009", "volume": "30", "issue": "3", "section": "", "partNumber": "", "partTitle": "", "pages": "293-299", "series": "", "seriesTitle": "", "seriesText": "", "journalAbbreviation": "Hum. Mutat.", "DOI": "10.1002/humu.20918", "citationKey": "", "url": "http://onlinelibrary.wiley.com.ccl.idm.oclc.org/doi/10.1002/humu.20918/abstract", "accessDate": "2015-11-29T06:35:13Z", "PMID": "", "PMCID": "", "ISSN": "1098-1004", "archive": "", "archiveLocation": "", "shortTitle": "", "language": "en", "libraryCatalog": "Wiley Online Library", "callNumber": "", "rights": "© 2009 Wiley-Liss, Inc.", "extra": "", "tags": [ { "tag": "DMD", "type": 1 }, { "tag": "Duchenne Muscular Dystrophy", "type": 1 }, { "tag": "Dystrophin", "type": 1 }, { "tag": "antisense oligonucleotides", "type": 1 }, { "tag": "exon skipping", "type": 1 }, { "tag": "therapy", "type": 1 } ], "collections": [ "IT64SMGH" ], "relations": {}, "dateAdded": "2015-11-29T06:35:13Z", "dateModified": "2015-11-29T06:35:13Z" } }, { "key": "PNXAUUBX", "version": 36, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/PNXAUUBX", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/PNXAUUBX", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/NZWUSMU5", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2651886, "username": "Mandar Makwana", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/mandar_makwana", "type": "text/html" } } }, "numChildren": 0 }, "data": { "key": "PNXAUUBX", "version": 36, "parentItem": "NZWUSMU5", "itemType": "attachment", "linkMode": "imported_url", "title": "PubMed Central Full Text PDF", "accessDate": "2015-11-29T05:58:28Z", "url": "http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2644416/pdf/nihms-92845.pdf", "note": "", "contentType": "application/pdf", "charset": "", "filename": "Parsons et al. - 2007 - Genetic Disruption of Calcineurin Improves Skeleta.pdf", "md5": "46cfa0519b16ffb34951d299b4c9ea0d", "mtime": 1448776745885, "tags": [], "relations": {}, "dateAdded": "2015-11-29T05:58:28Z", "dateModified": "2015-11-29T05:59:06Z" } }, { "key": "4QNCE8R9", "version": 34, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/4QNCE8R9", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/4QNCE8R9", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/NZWUSMU5", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2651886, "username": "Mandar Makwana", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/mandar_makwana", "type": "text/html" } } } }, "data": { "key": "4QNCE8R9", "version": 34, "parentItem": "NZWUSMU5", "itemType": "attachment", "linkMode": "linked_url", "title": "PubMed Central Link", "accessDate": "2015-11-29T05:58:28Z", "url": "http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2644416/", "note": "", "contentType": "text/html", "charset": "", "tags": [], "relations": {}, "dateAdded": "2015-11-29T05:58:28Z", "dateModified": "2015-11-29T05:58:28Z" } }, { "key": "NZWUSMU5", "version": 34, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/NZWUSMU5", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/NZWUSMU5", "type": "text/html" } }, "meta": { "createdByUser": { "id": 2651886, "username": "Mandar Makwana", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/mandar_makwana", "type": "text/html" } } }, "creatorSummary": "Parsons et al.", "parsedDate": "2007-03-30", "numChildren": 2 }, "data": { "key": "NZWUSMU5", "version": 34, "itemType": "journalArticle", "title": "Genetic Disruption of Calcineurin Improves Skeletal Muscle Pathology and Cardiac Disease in a Mouse Model of Limb-Girdle Muscular Dystrophy", "creators": [ { "creatorType": "author", "firstName": "Stephanie A.", "lastName": "Parsons" }, { "creatorType": "author", "firstName": "Douglas P.", "lastName": "Millay" }, { "creatorType": "author", "firstName": "Michelle A.", "lastName": "Sargent" }, { "creatorType": "author", "firstName": "Francisco J.", "lastName": "Naya" }, { "creatorType": "author", "firstName": "Elizabeth M.", "lastName": "McNally" }, { "creatorType": "author", "firstName": "H. Lee", "lastName": "Sweeney" }, { "creatorType": "author", "firstName": "Jeffery D.", "lastName": "Molkentin" } ], "abstractNote": "Calcineurin (Cn) is a Ca2+/calmodulin-dependent serine/threonine phosphatase that regulates differentiation-specific gene expression in diverse tissues, including the control of fiber-type switching in skeletal muscle. Recent studies have implicated Cn signaling in diminishing skeletal muscle pathogenesis associated with muscle injury or disease-related muscle degeneration. For example, use of the Cn inhibitor cyclosporine A has been shown to delay muscle regeneration following toxin-induced injury and inhibit regeneration in the dystrophin-deficient mdx mouse model of Duchenne muscular dystrophy. In contrast, transgenic expression of an activated mutant of Cn in skeletal muscle was shown to increase utrophin expression and reduce overall disease pathology in mdx mice. Here we examine the effect of altered Cn activation in the context of the δ-sarcoglycan-null (scgd−/−) mouse model of limb-girdle muscular dystrophy. In contrast to results discussed in mdx mice, genetic deletion of a loxP-targeted calcineurin B1 (CnB1) gene using a skeletal muscle-specific cre allele in the scgd−/− background substantially reduced skeletal muscle degeneration and histopathology compared with the scgd−/− genotype alone. A similar regression in scgd-dependent disease manifestation was also observed in calcineurin Aβ (CnAβ) gene-targeted mice in both skeletal muscle and heart. Conversely, increased Cn expression using a muscle-specific transgene increased cardiac fibrosis, decreased cardiac ventricular shortening, and increased muscle fiber loss in the quadriceps. Our results suggest that inhibition of Cn may benefit select types of muscular dystrophy.", "publicationTitle": "The Journal of biological chemistry", "publisher": "", "place": "", "date": "2007-3-30", "volume": "282", "issue": "13", "section": "", "partNumber": "", "partTitle": "", "pages": "10068-10078", "series": "", "seriesTitle": "", "seriesText": "", "journalAbbreviation": "J Biol Chem", "DOI": "10.1074/jbc.M609368200", "citationKey": "", "url": "http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2644416/", "accessDate": "2015-11-29T05:58:28Z", "PMID": "", "PMCID": "", "ISSN": "0021-9258", "archive": "", "archiveLocation": "", "shortTitle": "", "language": "", "libraryCatalog": "PubMed Central", "callNumber": "", "rights": "", "extra": "PMID: 17289669\nPMCID: PMC2644416", "tags": [], "collections": [ "MVN9GAN5" ], "relations": {}, "dateAdded": "2015-11-29T05:58:28Z", "dateModified": "2015-11-29T05:58:28Z" } }, { "key": "3SZN2N5P", "version": 35, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/3SZN2N5P", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/3SZN2N5P", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/8CDJNBSR", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2651886, "username": "Mandar Makwana", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/mandar_makwana", "type": "text/html" } } }, "numChildren": 0 }, "data": { "key": "3SZN2N5P", "version": 35, "parentItem": "8CDJNBSR", "itemType": "attachment", "linkMode": "imported_url", "title": "Full Text PDF", "accessDate": "2015-11-29T05:53:32Z", "url": "http://circres.ahajournals.org/content/94/8/1023.full.pdf", "note": "", "contentType": "application/pdf", "charset": "", "filename": "Lapidos et al. - 2004 - The Dystrophin Glycoprotein Complex Signaling Stre.pdf", "md5": "5cf6ab3fa664633cadb4dccb42217047", "mtime": 1448776436808, "tags": [], "relations": {}, "dateAdded": "2015-11-29T05:53:32Z", "dateModified": "2015-11-29T05:53:56Z" } }, { "key": "NUHUMW8M", "version": 35, "library": { "type": "group", "id": 434526, "name": "ALS 333 DMD Project", "links": { "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project", "type": "text/html" } } }, "links": { "self": { "href": "https://api.zotero.org/groups/434526/items/NUHUMW8M", "type": "application/json" }, "alternate": { "href": "https://www.zotero.org/groups/als_333_dmd_project/items/NUHUMW8M", "type": "text/html" }, "up": { "href": "https://api.zotero.org/groups/434526/items/SXQUDPHC", "type": "application/json" } }, "meta": { "createdByUser": { "id": 2651886, "username": "Mandar Makwana", "name": "", "links": { "alternate": { "href": "https://www.zotero.org/mandar_makwana", "type": "text/html" } } }, "numChildren": 0 }, "data": { "key": "NUHUMW8M", "version": 35, "parentItem": "SXQUDPHC", "itemType": "attachment", "linkMode": "imported_url", "title": "Full Text PDF", "accessDate": "2015-11-29T05:53:28Z", "url": "http://circres.ahajournals.org/content/94/8/1023.full.pdf", "note": "", "contentType": "application/pdf", "charset": "", "filename": "Lapidos et al. - 2004 - The Dystrophin Glycoprotein Complex Signaling Stre.pdf", "md5": "3bb840dc0958e3eab6958e425641c438", "mtime": 1448776435484, "tags": [], "relations": {}, "dateAdded": "2015-11-29T05:53:28Z", "dateModified": "2015-11-29T05:53:55Z" } } ]