Gene Transfer into the Mouse Retina Mediated by an Adeno-Associated Viral Vector
Gene transfer to photoreceptor cells may provide a means for arresting the retinal degeneration that is characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and...
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| Veröffentlicht in: | Human molecular genetics 1996-05, Vol.5 (5), p.591-594 |
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| container_end_page | 594 |
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| container_issue | 5 |
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| container_title | Human molecular genetics |
| container_volume | 5 |
| creator | Ali, Robin R. Reichel, Martin B. Thrasher, Adrian J. Levinsky, Roland J. Kinnon, Christine Kanuga, Naheed Hunt, David M. Bhattacharya, Shomi S. |
| description | Gene transfer to photoreceptor cells may provide a means for arresting the retinal degeneration that is characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and immune responses directed against vector-specific proteins. An alternative vector system based on adeno-associated virus (AAV) may obviate these problems, and may be useful for transduction of neuronal cells. In this study we have demonstrated successful transduction of all layers of the neuroretina as well as the retinal pigment epithelium (RPE) following subretinal injection of recombinant AAV particles encoding lac Z. Furthermore, the efficiency of transduction of photoreceptors is significantly higher than that achieved with an equivalent adenoviral vector. This is the first report showing that AAV is capable of transducing photoreceptor cells and supports the use of this vector system for gene therapy of retinal diseases such as RP. |
| doi_str_mv | 10.1093/hmg/5.5.591 |
| format | Article |
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However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and immune responses directed against vector-specific proteins. An alternative vector system based on adeno-associated virus (AAV) may obviate these problems, and may be useful for transduction of neuronal cells. In this study we have demonstrated successful transduction of all layers of the neuroretina as well as the retinal pigment epithelium (RPE) following subretinal injection of recombinant AAV particles encoding lac Z. Furthermore, the efficiency of transduction of photoreceptors is significantly higher than that achieved with an equivalent adenoviral vector. This is the first report showing that AAV is capable of transducing photoreceptor cells and supports the use of this vector system for gene therapy of retinal diseases such as RP.</description><identifier>ISSN: 0964-6906</identifier><identifier>ISSN: 1460-2083</identifier><identifier>EISSN: 1460-2083</identifier><identifier>DOI: 10.1093/hmg/5.5.591</identifier><identifier>PMID: 8733124</identifier><language>eng</language><publisher>Oxford: Oxford University Press</publisher><subject>adeno-associated virus ; Animals ; Base Sequence ; Biological and medical sciences ; Biotechnology ; Dependovirus - genetics ; DNA Primers ; Fundamental and applied biological sciences. Psychology ; Gene therapy ; Gene Transfer Techniques ; Genes, Reporter ; Genetic Vectors ; Health. Pharmaceutical industry ; Industrial applications and implications. Economical aspects ; Lac Operon ; Mice ; Mice, Inbred BALB C ; Mice, Nude ; Molecular Sequence Data ; Pigment Epithelium of Eye - ultrastructure ; Retina - metabolism ; Retinitis Pigmentosa - genetics ; Transfection</subject><ispartof>Human molecular genetics, 1996-05, Vol.5 (5), p.591-594</ispartof><rights>1996 INIST-CNRS</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c419t-9785575f9f38b2fa7890fa06173fc345f701ca5c3fd3a5d359d4704db007d1483</citedby></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=3070054$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/8733124$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Ali, Robin R.</creatorcontrib><creatorcontrib>Reichel, Martin B.</creatorcontrib><creatorcontrib>Thrasher, Adrian J.</creatorcontrib><creatorcontrib>Levinsky, Roland J.</creatorcontrib><creatorcontrib>Kinnon, Christine</creatorcontrib><creatorcontrib>Kanuga, Naheed</creatorcontrib><creatorcontrib>Hunt, David M.</creatorcontrib><creatorcontrib>Bhattacharya, Shomi S.</creatorcontrib><title>Gene Transfer into the Mouse Retina Mediated by an Adeno-Associated Viral Vector</title><title>Human molecular genetics</title><addtitle>Human Molecular Genetics</addtitle><description>Gene transfer to photoreceptor cells may provide a means for arresting the retinal degeneration that is characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and immune responses directed against vector-specific proteins. An alternative vector system based on adeno-associated virus (AAV) may obviate these problems, and may be useful for transduction of neuronal cells. In this study we have demonstrated successful transduction of all layers of the neuroretina as well as the retinal pigment epithelium (RPE) following subretinal injection of recombinant AAV particles encoding lac Z. Furthermore, the efficiency of transduction of photoreceptors is significantly higher than that achieved with an equivalent adenoviral vector. This is the first report showing that AAV is capable of transducing photoreceptor cells and supports the use of this vector system for gene therapy of retinal diseases such as RP.</description><subject>adeno-associated virus</subject><subject>Animals</subject><subject>Base Sequence</subject><subject>Biological and medical sciences</subject><subject>Biotechnology</subject><subject>Dependovirus - genetics</subject><subject>DNA Primers</subject><subject>Fundamental and applied biological sciences. Psychology</subject><subject>Gene therapy</subject><subject>Gene Transfer Techniques</subject><subject>Genes, Reporter</subject><subject>Genetic Vectors</subject><subject>Health. Pharmaceutical industry</subject><subject>Industrial applications and implications. Economical aspects</subject><subject>Lac Operon</subject><subject>Mice</subject><subject>Mice, Inbred BALB C</subject><subject>Mice, Nude</subject><subject>Molecular Sequence Data</subject><subject>Pigment Epithelium of Eye - ultrastructure</subject><subject>Retina - metabolism</subject><subject>Retinitis Pigmentosa - genetics</subject><subject>Transfection</subject><issn>0964-6906</issn><issn>1460-2083</issn><issn>1460-2083</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>1996</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNo9kE1LAzEQhoMoWqsnz0IO4kW2TppkszkWv8Wi-FHES0iTia5udzXZgv57t7SUOQzM-_AyPIQcMBgw0Pz0Y_Z-KgfdaLZBekzkkA2h4JukBzoXWa4h3yG7KX0CsFxwtU22C8U5G4oeebjCGulztHUKGGlZtw1tP5COm3lC-ohtWVs6Rl_aFj2d_lFb05HHuslGKTVueZ6U0VZ0gq5t4h7ZCrZKuL_affJyefF8dp3d3V_dnI3uMieYbjOtCimVDDrwYjoMVhUagoWcKR4cFzIoYM5Kx4PnVnoutRcKhJ8CKM9EwfvkeNn7HZufOabWzMrksKpsjd3vhsmcCaVVB54sQReblCIG8x3LmY1_hoFZ-DOdPyMXo1lHH65q59MZ-jW7EtblR6vcJmer0IlzZVpjHBSAXGDZEitTi7_r2MYvkyuupLl-fTPnxUSfP92OzSv_B3Mmhek</recordid><startdate>19960501</startdate><enddate>19960501</enddate><creator>Ali, Robin R.</creator><creator>Reichel, Martin B.</creator><creator>Thrasher, Adrian J.</creator><creator>Levinsky, Roland J.</creator><creator>Kinnon, Christine</creator><creator>Kanuga, Naheed</creator><creator>Hunt, David M.</creator><creator>Bhattacharya, Shomi S.</creator><general>Oxford University Press</general><scope>BSCLL</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7QO</scope><scope>8FD</scope><scope>FR3</scope><scope>P64</scope><scope>RC3</scope></search><sort><creationdate>19960501</creationdate><title>Gene Transfer into the Mouse Retina Mediated by an Adeno-Associated Viral Vector</title><author>Ali, Robin R. ; Reichel, Martin B. ; Thrasher, Adrian J. ; Levinsky, Roland J. ; Kinnon, Christine ; Kanuga, Naheed ; Hunt, David M. ; Bhattacharya, Shomi S.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c419t-9785575f9f38b2fa7890fa06173fc345f701ca5c3fd3a5d359d4704db007d1483</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>1996</creationdate><topic>adeno-associated virus</topic><topic>Animals</topic><topic>Base Sequence</topic><topic>Biological and medical sciences</topic><topic>Biotechnology</topic><topic>Dependovirus - genetics</topic><topic>DNA Primers</topic><topic>Fundamental and applied biological sciences. Psychology</topic><topic>Gene therapy</topic><topic>Gene Transfer Techniques</topic><topic>Genes, Reporter</topic><topic>Genetic Vectors</topic><topic>Health. Pharmaceutical industry</topic><topic>Industrial applications and implications. Economical aspects</topic><topic>Lac Operon</topic><topic>Mice</topic><topic>Mice, Inbred BALB C</topic><topic>Mice, Nude</topic><topic>Molecular Sequence Data</topic><topic>Pigment Epithelium of Eye - ultrastructure</topic><topic>Retina - metabolism</topic><topic>Retinitis Pigmentosa - genetics</topic><topic>Transfection</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Ali, Robin R.</creatorcontrib><creatorcontrib>Reichel, Martin B.</creatorcontrib><creatorcontrib>Thrasher, Adrian J.</creatorcontrib><creatorcontrib>Levinsky, Roland J.</creatorcontrib><creatorcontrib>Kinnon, Christine</creatorcontrib><creatorcontrib>Kanuga, Naheed</creatorcontrib><creatorcontrib>Hunt, David M.</creatorcontrib><creatorcontrib>Bhattacharya, Shomi S.</creatorcontrib><collection>Istex</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Biotechnology Research Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><jtitle>Human molecular genetics</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Ali, Robin R.</au><au>Reichel, Martin B.</au><au>Thrasher, Adrian J.</au><au>Levinsky, Roland J.</au><au>Kinnon, Christine</au><au>Kanuga, Naheed</au><au>Hunt, David M.</au><au>Bhattacharya, Shomi S.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Gene Transfer into the Mouse Retina Mediated by an Adeno-Associated Viral Vector</atitle><jtitle>Human molecular genetics</jtitle><addtitle>Human Molecular Genetics</addtitle><date>1996-05-01</date><risdate>1996</risdate><volume>5</volume><issue>5</issue><spage>591</spage><epage>594</epage><pages>591-594</pages><issn>0964-6906</issn><issn>1460-2083</issn><eissn>1460-2083</eissn><abstract>Gene transfer to photoreceptor cells may provide a means for arresting the retinal degeneration that is characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and immune responses directed against vector-specific proteins. An alternative vector system based on adeno-associated virus (AAV) may obviate these problems, and may be useful for transduction of neuronal cells. In this study we have demonstrated successful transduction of all layers of the neuroretina as well as the retinal pigment epithelium (RPE) following subretinal injection of recombinant AAV particles encoding lac Z. Furthermore, the efficiency of transduction of photoreceptors is significantly higher than that achieved with an equivalent adenoviral vector. This is the first report showing that AAV is capable of transducing photoreceptor cells and supports the use of this vector system for gene therapy of retinal diseases such as RP.</abstract><cop>Oxford</cop><pub>Oxford University Press</pub><pmid>8733124</pmid><doi>10.1093/hmg/5.5.591</doi><tpages>4</tpages><oa>free_for_read</oa></addata></record> |
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| identifier | ISSN: 0964-6906 |
| ispartof | Human molecular genetics, 1996-05, Vol.5 (5), p.591-594 |
| issn | 0964-6906 1460-2083 1460-2083 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_15614797 |
| source | MEDLINE; Oxford University Press Journals All Titles (1996-Current); EZB-FREE-00999 freely available EZB journals |
| subjects | adeno-associated virus Animals Base Sequence Biological and medical sciences Biotechnology Dependovirus - genetics DNA Primers Fundamental and applied biological sciences. Psychology Gene therapy Gene Transfer Techniques Genes, Reporter Genetic Vectors Health. Pharmaceutical industry Industrial applications and implications. Economical aspects Lac Operon Mice Mice, Inbred BALB C Mice, Nude Molecular Sequence Data Pigment Epithelium of Eye - ultrastructure Retina - metabolism Retinitis Pigmentosa - genetics Transfection |
| title | Gene Transfer into the Mouse Retina Mediated by an Adeno-Associated Viral Vector |
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