Gene therapies in canine models for Duchenne muscular dystrophy
Therapies for Duchenne muscular dystrophy (DMD) must first be tested in animal models to determine proof-of-concept, efficacy, and importantly, safety. The murine and canine models for DMD are genetically homologous and most commonly used in pre-clinical testing. Although the mouse is a strong, proo...
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| Veröffentlicht in: | Human genetics 2019-05, Vol.138 (5), p.483-489 |
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| container_title | Human genetics |
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| creator | Nghiem, Peter P. Kornegay, Joe N. |
| description | Therapies for Duchenne muscular dystrophy (DMD) must first be tested in animal models to determine proof-of-concept, efficacy, and importantly, safety. The murine and canine models for DMD are genetically homologous and most commonly used in pre-clinical testing. Although the mouse is a strong, proof-of-concept model, affected dogs show more analogous clinical and immunological disease progression compared to boys with DMD. As such, evaluating genetic therapies in the canine models may better predict response at the genetic, phenotypic, and immunological levels. We review the use of canine models for DMD and their benefits as it pertains to genetic therapy studies, including gene replacement, exon skipping, and gene editing. |
| doi_str_mv | 10.1007/s00439-019-01976-z |
| format | Article |
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The murine and canine models for DMD are genetically homologous and most commonly used in pre-clinical testing. Although the mouse is a strong, proof-of-concept model, affected dogs show more analogous clinical and immunological disease progression compared to boys with DMD. As such, evaluating genetic therapies in the canine models may better predict response at the genetic, phenotypic, and immunological levels. We review the use of canine models for DMD and their benefits as it pertains to genetic therapy studies, including gene replacement, exon skipping, and gene editing.</description><identifier>ISSN: 0340-6717</identifier><identifier>EISSN: 1432-1203</identifier><identifier>DOI: 10.1007/s00439-019-01976-z</identifier><identifier>PMID: 30734120</identifier><language>eng</language><publisher>Berlin/Heidelberg: Springer Berlin Heidelberg</publisher><subject>Animal models ; Biomedical and Life Sciences ; Biomedicine ; Canine Genetics ; Dogs ; Duchenne muscular dystrophy ; Duchenne's muscular dystrophy ; Exon skipping ; Gene Function ; Gene therapy ; Genes ; Genome editing ; Human Genetics ; Immunological diseases ; Immunology ; Metabolic Diseases ; Molecular Medicine ; Muscular dystrophy ; Retirement benefits ; Review</subject><ispartof>Human genetics, 2019-05, Vol.138 (5), p.483-489</ispartof><rights>Springer-Verlag GmbH Germany, part of Springer Nature 2019</rights><rights>COPYRIGHT 2019 Springer</rights><rights>Human Genetics is a copyright of Springer, (2019). All Rights Reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c476t-7bb2caf0a4fce4d8507a3ead54655f5f58aefa2f8fb9202149eb1574ef78f85c3</citedby><cites>FETCH-LOGICAL-c476t-7bb2caf0a4fce4d8507a3ead54655f5f58aefa2f8fb9202149eb1574ef78f85c3</cites><orcidid>0000-0002-8796-8123</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s00439-019-01976-z$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s00439-019-01976-z$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,776,780,27901,27902,41464,42533,51294</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/30734120$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Nghiem, Peter P.</creatorcontrib><creatorcontrib>Kornegay, Joe N.</creatorcontrib><title>Gene therapies in canine models for Duchenne muscular dystrophy</title><title>Human genetics</title><addtitle>Hum Genet</addtitle><addtitle>Hum Genet</addtitle><description>Therapies for Duchenne muscular dystrophy (DMD) must first be tested in animal models to determine proof-of-concept, efficacy, and importantly, safety. 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We review the use of canine models for DMD and their benefits as it pertains to genetic therapy studies, including gene replacement, exon skipping, and gene editing.</description><subject>Animal models</subject><subject>Biomedical and Life Sciences</subject><subject>Biomedicine</subject><subject>Canine Genetics</subject><subject>Dogs</subject><subject>Duchenne muscular dystrophy</subject><subject>Duchenne's muscular dystrophy</subject><subject>Exon skipping</subject><subject>Gene Function</subject><subject>Gene therapy</subject><subject>Genes</subject><subject>Genome editing</subject><subject>Human Genetics</subject><subject>Immunological diseases</subject><subject>Immunology</subject><subject>Metabolic Diseases</subject><subject>Molecular Medicine</subject><subject>Muscular dystrophy</subject><subject>Retirement benefits</subject><subject>Review</subject><issn>0340-6717</issn><issn>1432-1203</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2019</creationdate><recordtype>article</recordtype><sourceid>BENPR</sourceid><recordid>eNp9kUtr3DAUhUVpaCZp_0AXxdBNs3B69bLsVQlpmgYChT7WQpavZhxsaSrZkMmvjyaTNkwJ5SIER9856HIIeUvhlAKojwlA8KYE-nBUVd69IAsqOCspA_6SLIALKCtF1SE5SukGgMqGyVfkkIPiIkML8ukSPRbTCqNZ95iK3hfW-D5rY-hwSIULsfg82xX6rTYnOw8mFt0mTTGsV5vX5MCZIeGbx_uY_Ppy8fP8a3n97fLq_Oy6tEJVU6nallnjwAhnUXS1BGU4mk6KSkqXpzboDHO1axsGjIoGWyqVQKdqV0vLj8mHXe46ht8zpkmPfbI4DMZjmJNmtBaUy6apMvr-H_QmzNHn32VKKaAKmvqJWpoBde9dmKKx21B9Juuq4ZSrLXX6DJWnw7G3waPrs75nONkzZGbC22lp5pT01Y_v-yzbsTaGlCI6vY79aOJGU9DbhvWuYZ3b1Q8N67tseve43dyO2P21_Kk0A3wHpPzklxif1v9P7D0J3K7Z</recordid><startdate>20190501</startdate><enddate>20190501</enddate><creator>Nghiem, Peter P.</creator><creator>Kornegay, Joe N.</creator><general>Springer Berlin Heidelberg</general><general>Springer</general><general>Springer Nature B.V</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>ISR</scope><scope>3V.</scope><scope>7QP</scope><scope>7TK</scope><scope>7TM</scope><scope>7X7</scope><scope>7XB</scope><scope>88A</scope><scope>88E</scope><scope>8AO</scope><scope>8C1</scope><scope>8FD</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M7P</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>RC3</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0002-8796-8123</orcidid></search><sort><creationdate>20190501</creationdate><title>Gene therapies in canine models for Duchenne muscular dystrophy</title><author>Nghiem, Peter P. ; 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The murine and canine models for DMD are genetically homologous and most commonly used in pre-clinical testing. Although the mouse is a strong, proof-of-concept model, affected dogs show more analogous clinical and immunological disease progression compared to boys with DMD. As such, evaluating genetic therapies in the canine models may better predict response at the genetic, phenotypic, and immunological levels. We review the use of canine models for DMD and their benefits as it pertains to genetic therapy studies, including gene replacement, exon skipping, and gene editing.</abstract><cop>Berlin/Heidelberg</cop><pub>Springer Berlin Heidelberg</pub><pmid>30734120</pmid><doi>10.1007/s00439-019-01976-z</doi><tpages>7</tpages><orcidid>https://orcid.org/0000-0002-8796-8123</orcidid></addata></record> |
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| subjects | Animal models Biomedical and Life Sciences Biomedicine Canine Genetics Dogs Duchenne muscular dystrophy Duchenne's muscular dystrophy Exon skipping Gene Function Gene therapy Genes Genome editing Human Genetics Immunological diseases Immunology Metabolic Diseases Molecular Medicine Muscular dystrophy Retirement benefits Review |
| title | Gene therapies in canine models for Duchenne muscular dystrophy |
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