Recombinant adeno-associated virus vectors for gene therapy
Recombinant adeno-associated virus (rAAV) vectors are based on a non-pathogenic human parvovirus (AAV) that is unique in its ability to persist in human cells without causing any pathologic effects. Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led...
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| Veröffentlicht in: | Expert opinion on biological therapy 2004-07, Vol.4 (7), p.1093-1101 |
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| container_title | Expert opinion on biological therapy |
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| creator | Conlon, Thomas J Flotte, Terence R |
| description | Recombinant adeno-associated virus (rAAV) vectors are based on a non-pathogenic human parvovirus (AAV) that is unique in its ability to persist in human cells without causing any pathologic effects. Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led to an understanding of the mechanisms of cell attachment and entry, cytoplasmic translocation, nuclear entry, conversion to active double-stranded DNA, activation of transcription and establishment of persistent molecular forms. Each of these areas is individually discussed, as are recent applications in vivo in preclinical models and clinical trials. |
| doi_str_mv | 10.1517/14712598.4.7.1093 |
| format | Article |
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Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led to an understanding of the mechanisms of cell attachment and entry, cytoplasmic translocation, nuclear entry, conversion to active double-stranded DNA, activation of transcription and establishment of persistent molecular forms. Each of these areas is individually discussed, as are recent applications in vivo in preclinical models and clinical trials.</description><subject>Adeno-associated virus</subject><subject>Animals</subject><subject>Biological Transport</subject><subject>Capsid - ultrastructure</subject><subject>Clinical Trials as Topic</subject><subject>Dependovirus - genetics</subject><subject>Dependovirus - pathogenicity</subject><subject>Dependovirus - ultrastructure</subject><subject>DNA, Recombinant - genetics</subject><subject>DNA, Single-Stranded - genetics</subject><subject>DNA, Viral - genetics</subject><subject>Gene Expression Regulation, Viral</subject><subject>gene therapy</subject><subject>Genes, Synthetic</subject><subject>Genetic Therapy - methods</subject><subject>Genetic Vectors - administration & dosage</subject><subject>Genetic Vectors - genetics</subject><subject>Genetic Vectors - therapeutic use</subject><subject>Humans</subject><subject>Injections, Intramuscular</subject><subject>Muscle Fibers, Skeletal - virology</subject><subject>Mutagenesis, Insertional</subject><subject>Parvovirus</subject><subject>Receptors, Virus - physiology</subject><subject>Transduction, Genetic</subject><subject>viral vectors</subject><issn>1471-2598</issn><issn>1744-7682</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2004</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kE1LxDAQhoMofv8AL9KTt66ZJG1a9CLiFwiC6DlM06lbaZs1aVf23xvZFfHiaTLkeV-Gh7ET4DPIQJ-D0iCyspipmZ4BL-UW2wetVKrzQmzHd_xPv4E9dhDCO-eCl5nYZXuQibzIdb7PLp7Jur5qBxzGBGsaXIohONviSHWybP0UkiXZ0fmQNM4nbzRQMs7J42J1xHYa7AIdb-Yhe729ebm-Tx-f7h6urx5Tq6QcUwkgZJY1VFRaYK7qSoKVudA1NGVTyUKVgABos6pSohCSOIosJ1uQzqVAecjO1r0L7z4mCqPp22Cp63AgNwUjuOSgCh5BWIPWuxA8NWbh2x79ygA338bMjzGjTFyisZg53ZRPVU_1b2KjKAKXa6AdooAeP53vajPiqnO-8TjYNhj5X__Fn_icsBvnFj2Zdzf5IYr757ovamyMPA</recordid><startdate>20040701</startdate><enddate>20040701</enddate><creator>Conlon, Thomas J</creator><creator>Flotte, Terence R</creator><general>Ashley Publications Ltd</general><general>Taylor & Francis</general><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>7U9</scope><scope>8FD</scope><scope>FR3</scope><scope>H94</scope><scope>P64</scope><scope>RC3</scope></search><sort><creationdate>20040701</creationdate><title>Recombinant adeno-associated virus vectors for gene therapy</title><author>Conlon, Thomas J ; 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| fulltext | fulltext |
| identifier | ISSN: 1471-2598 |
| ispartof | Expert opinion on biological therapy, 2004-07, Vol.4 (7), p.1093-1101 |
| issn | 1471-2598 1744-7682 |
| language | eng |
| recordid | cdi_pubmed_primary_15268676 |
| source | MEDLINE; Taylor & Francis Medical Library - CRKN; Taylor & Francis Journals Complete |
| subjects | Adeno-associated virus Animals Biological Transport Capsid - ultrastructure Clinical Trials as Topic Dependovirus - genetics Dependovirus - pathogenicity Dependovirus - ultrastructure DNA, Recombinant - genetics DNA, Single-Stranded - genetics DNA, Viral - genetics Gene Expression Regulation, Viral gene therapy Genes, Synthetic Genetic Therapy - methods Genetic Vectors - administration & dosage Genetic Vectors - genetics Genetic Vectors - therapeutic use Humans Injections, Intramuscular Muscle Fibers, Skeletal - virology Mutagenesis, Insertional Parvovirus Receptors, Virus - physiology Transduction, Genetic viral vectors |
| title | Recombinant adeno-associated virus vectors for gene therapy |
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