Gene therapy for Parkinson's disease.
Identifieur interne : 001402 ( PubMed/Checkpoint ); précédent : 001401; suivant : 001403Gene therapy for Parkinson's disease.
Auteurs : P. Horellou [France] ; J. MalletSource :
- Molecular neurobiology [ 0893-7648 ] ; 1997.
English descriptors
- KwdEn :
- Adenoviruses, Human (genetics), Animals, Astrocytes (metabolism), Astrocytes (transplantation), Cell Survival, Cell Transplantation, Cells, Cultured (transplantation), Corpus Striatum (metabolism), Corpus Striatum (pathology), Dependovirus (genetics), Dopamine (metabolism), Fibroblasts (metabolism), Fibroblasts (transplantation), Genetic Therapy, Genetic Vectors (administration & dosage), Genetic Vectors (genetics), Glial Cell Line-Derived Neurotrophic Factor, Humans, Nerve Degeneration, Nerve Growth Factors, Nerve Tissue Proteins (genetics), Nerve Tissue Proteins (metabolism), Neurons (pathology), Oxidopamine (toxicity), Parkinson Disease (metabolism), Parkinson Disease (therapy), Parkinson Disease, Secondary (chemically induced), Parkinson Disease, Secondary (therapy), Rats, Recombinant Fusion Proteins (metabolism), Retroviridae (genetics), Stem Cell Transplantation, Stem Cells (metabolism), Tyrosine 3-Monooxygenase (genetics), Tyrosine 3-Monooxygenase (metabolism).
- MESH :
- chemical , genetics : Nerve Tissue Proteins, Tyrosine 3-Monooxygenase.
- chemical , metabolism : Dopamine, Nerve Tissue Proteins, Recombinant Fusion Proteins, Tyrosine 3-Monooxygenase.
- administration & dosage : Genetic Vectors.
- chemically induced : Parkinson Disease, Secondary.
- genetics : Adenoviruses, Human, Dependovirus, Genetic Vectors, Retroviridae.
- metabolism : Astrocytes, Corpus Striatum, Fibroblasts, Parkinson Disease, Stem Cells.
- pathology : Corpus Striatum, Neurons.
- therapy : Parkinson Disease, Parkinson Disease, Secondary.
- chemical , toxicity : Oxidopamine.
- transplantation : Astrocytes, Cells, Cultured, Fibroblasts.
- Animals, Cell Survival, Cell Transplantation, Genetic Therapy, Glial Cell Line-Derived Neurotrophic Factor, Humans, Nerve Degeneration, Nerve Growth Factors, Rats, Stem Cell Transplantation.
Abstract
Gene therapy is a potentially powerful approach to the treatment of neurological diseases. The discovery of neurotrophic factors inhibiting neurodegenerative processes and neurotransmitter-synthesizing enzymes provides the basis for current gene therapy strategies for Parkinson's disease. Genes can be transferred by viral or nonviral vectors. Of the various possible vectors, recombinant retroviruses are the most efficient for genetic modification of cells in vitro that can thereafter be used for transplantation (ex vivo gene therapy approach). Recently, in vivo gene transfer to the brain has been developed using adenovirus vectors. One of the advantages of recombinant adenovirus is that it can transduce both quiescent and actively dividing cells, thereby allowing both direct in vivo gene transfer and ex vivo gene transfer to neural cells. Probably because the brain is partially protected from the immune system, the expression of adenoviral vectors persists for several months with little inflammation. Novel therapeutic tools, such as vectors for gene therapy have to be evaluated in terms of efficacy and safety for future clinical trials. These vectors still need to be improved to allow long-term and possibly regulatable expression of the transgene.
DOI: 10.1007/BF02740636
PubMed: 9396012
Affiliations:
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pubmed:9396012Le document en format XML
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The discovery of neurotrophic factors inhibiting neurodegenerative processes and neurotransmitter-synthesizing enzymes provides the basis for current gene therapy strategies for Parkinson's disease. Genes can be transferred by viral or nonviral vectors. Of the various possible vectors, recombinant retroviruses are the most efficient for genetic modification of cells in vitro that can thereafter be used for transplantation (ex vivo gene therapy approach). Recently, in vivo gene transfer to the brain has been developed using adenovirus vectors. One of the advantages of recombinant adenovirus is that it can transduce both quiescent and actively dividing cells, thereby allowing both direct in vivo gene transfer and ex vivo gene transfer to neural cells. Probably because the brain is partially protected from the immune system, the expression of adenoviral vectors persists for several months with little inflammation. Novel therapeutic tools, such as vectors for gene therapy have to be evaluated in terms of efficacy and safety for future clinical trials. These vectors still need to be improved to allow long-term and possibly regulatable expression of the transgene.</div></front></TEI><pubmed><MedlineCitation Status="MEDLINE" Owner="NLM"><PMID Version="1">9396012</PMID><DateCreated><Year>1998</Year><Month>01</Month><Day>13</Day></DateCreated><DateCompleted><Year>1998</Year><Month>01</Month><Day>13</Day></DateCompleted><DateRevised><Year>2013</Year><Month>11</Month><Day>21</Day></DateRevised><Article PubModel="Print"><Journal><ISSN IssnType="Print">0893-7648</ISSN><JournalIssue CitedMedium="Print"><Volume>15</Volume><Issue>2</Issue><PubDate><Year>1997</Year><Month>Oct</Month></PubDate></JournalIssue><Title>Molecular neurobiology</Title><ISOAbbreviation>Mol. Neurobiol.</ISOAbbreviation></Journal><ArticleTitle>Gene therapy for Parkinson's disease.</ArticleTitle><Pagination><MedlinePgn>241-56</MedlinePgn></Pagination><Abstract><AbstractText>Gene therapy is a potentially powerful approach to the treatment of neurological diseases. The discovery of neurotrophic factors inhibiting neurodegenerative processes and neurotransmitter-synthesizing enzymes provides the basis for current gene therapy strategies for Parkinson's disease. Genes can be transferred by viral or nonviral vectors. Of the various possible vectors, recombinant retroviruses are the most efficient for genetic modification of cells in vitro that can thereafter be used for transplantation (ex vivo gene therapy approach). Recently, in vivo gene transfer to the brain has been developed using adenovirus vectors. One of the advantages of recombinant adenovirus is that it can transduce both quiescent and actively dividing cells, thereby allowing both direct in vivo gene transfer and ex vivo gene transfer to neural cells. Probably because the brain is partially protected from the immune system, the expression of adenoviral vectors persists for several months with little inflammation. Novel therapeutic tools, such as vectors for gene therapy have to be evaluated in terms of efficacy and safety for future clinical trials. These vectors still need to be improved to allow long-term and possibly regulatable expression of the transgene.</AbstractText></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Horellou</LastName><ForeName>P</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>C 9923 CNRS, Laboratoire de Génétique Moleculaire de la Neurotransmission et des Processus Dégénératifs, Hopital de la Pitié Salpêtriere, Bâtiment CERVI, Paris, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Mallet</LastName><ForeName>J</ForeName><Initials>J</Initials></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D013485">Research Support, Non-U.S. Gov't</PublicationType><PublicationType UI="D016454">Review</PublicationType></PublicationTypeList></Article><MedlineJournalInfo><Country>United States</Country><MedlineTA>Mol 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MajorTopicYN="N">Fibroblasts</DescriptorName><QualifierName UI="Q000378" MajorTopicYN="N">metabolism</QualifierName><QualifierName UI="Q000637" MajorTopicYN="N">transplantation</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D015316" MajorTopicYN="Y">Genetic Therapy</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D005822" MajorTopicYN="N">Genetic Vectors</DescriptorName><QualifierName UI="Q000008" MajorTopicYN="N">administration & dosage</QualifierName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D051100" MajorTopicYN="N">Glial Cell Line-Derived Neurotrophic Factor</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D006801" MajorTopicYN="N">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D009410" MajorTopicYN="N">Nerve Degeneration</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D009414" MajorTopicYN="Y">Nerve Growth Factors</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D009419" MajorTopicYN="N">Nerve Tissue Proteins</DescriptorName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName><QualifierName UI="Q000378" MajorTopicYN="Y">metabolism</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D009474" MajorTopicYN="N">Neurons</DescriptorName><QualifierName UI="Q000473" MajorTopicYN="N">pathology</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D016627" MajorTopicYN="N">Oxidopamine</DescriptorName><QualifierName UI="Q000633" MajorTopicYN="N">toxicity</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D010300" MajorTopicYN="N">Parkinson Disease</DescriptorName><QualifierName UI="Q000378" MajorTopicYN="N">metabolism</QualifierName><QualifierName UI="Q000628" MajorTopicYN="Y">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D010302" MajorTopicYN="N">Parkinson Disease, Secondary</DescriptorName><QualifierName UI="Q000139" MajorTopicYN="N">chemically induced</QualifierName><QualifierName UI="Q000628" MajorTopicYN="N">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D051381" MajorTopicYN="N">Rats</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D011993" MajorTopicYN="N">Recombinant Fusion Proteins</DescriptorName><QualifierName UI="Q000378" MajorTopicYN="N">metabolism</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D012190" MajorTopicYN="N">Retroviridae</DescriptorName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D033581" MajorTopicYN="N">Stem Cell Transplantation</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D013234" MajorTopicYN="N">Stem Cells</DescriptorName><QualifierName UI="Q000378" MajorTopicYN="N">metabolism</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D014446" MajorTopicYN="N">Tyrosine 3-Monooxygenase</DescriptorName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName><QualifierName UI="Q000378" MajorTopicYN="Y">metabolism</QualifierName></MeshHeading></MeshHeadingList><NumberOfReferences>93</NumberOfReferences></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="pubmed"><Year>1997</Year><Month>12</Month><Day>13</Day></PubMedPubDate><PubMedPubDate PubStatus="medline"><Year>1997</Year><Month>12</Month><Day>13</Day><Hour>0</Hour><Minute>1</Minute></PubMedPubDate><PubMedPubDate PubStatus="entrez"><Year>1997</Year><Month>12</Month><Day>13</Day><Hour>0</Hour><Minute>0</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="pubmed">9396012</ArticleId><ArticleId IdType="doi">10.1007/BF02740636</ArticleId></ArticleIdList></PubmedData></pubmed><affiliations><list><country><li>France</li></country><region><li>Île-de-France</li></region><settlement><li>Paris</li></settlement></list><tree><noCountry><name sortKey="Mallet, J" sort="Mallet, J" uniqKey="Mallet J" first="J" last="Mallet">J. Mallet</name></noCountry><country name="France"><region name="Île-de-France"><name sortKey="Horellou, P" sort="Horellou, P" uniqKey="Horellou P" first="P" last="Horellou">P. Horellou</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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