Introduction to gene therapy: a clinical aftermath.
Identifieur interne : 001564 ( Main/Exploration ); précédent : 001563; suivant : 001565Introduction to gene therapy: a clinical aftermath.
Auteurs : Patrice P. Denèfle [France]Source :
- Methods in molecular biology (Clifton, N.J.) [ 1940-6029 ] ; 2011.
English descriptors
- KwdEn :
- Animals, Clinical Trials as Topic, Cystic Fibrosis (genetics), Cystic Fibrosis (therapy), Genetic Therapy (legislation & jurisprudence), Genetic Therapy (methods), Genetic Therapy (trends), Genetic Vectors (therapeutic use), Humans, Lipoprotein Lipase (genetics), Lipoprotein Lipase (therapeutic use), Muscular Dystrophy, Duchenne (genetics), Muscular Dystrophy, Duchenne (therapy), Parkinson Disease (genetics), Parkinson Disease (therapy), Peripheral Vascular Diseases (genetics), Peripheral Vascular Diseases (therapy), Severe Combined Immunodeficiency (therapy).
- MESH :
- chemical , genetics : Lipoprotein Lipase.
- genetics : Cystic Fibrosis, Muscular Dystrophy, Duchenne, Parkinson Disease, Peripheral Vascular Diseases.
- legislation & jurisprudence : Genetic Therapy.
- methods : Genetic Therapy.
- therapeutic use : Genetic Vectors, Lipoprotein Lipase.
- therapy : Cystic Fibrosis, Muscular Dystrophy, Duchenne, Parkinson Disease, Peripheral Vascular Diseases, Severe Combined Immunodeficiency.
- trends : Genetic Therapy.
- Animals, Clinical Trials as Topic, Humans.
Abstract
Despite three decades of huge progress in molecular genetics, in cloning of disease causative gene as well as technology breakthroughs in viral biotechnology, out of thousands of gene therapy clinical trials that have been initiated, only very few are now reaching regulatory approval. We shall review some of the major hurdles, and based on the current either positive or negative examples, we try to initiate drawing a learning curve from experience and possibly identify the major drivers for future successful achievement of human gene therapy trials.
DOI: 10.1007/978-1-61779-095-9_2
PubMed: 21590392
Affiliations:
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Le document en format XML
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Denèfle</name><affiliation wicri:level="3"><nlm:affiliation>Translational Sciences, IPSEN, and Biotherapies, ParisTech Institute, Paris-Descartes University, Paris, France. pdenefle@gmail.com</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Translational Sciences, IPSEN, and Biotherapies, ParisTech Institute, Paris-Descartes University, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2011">2011</date><idno type="RBID">pubmed:21590392</idno><idno type="pmid">21590392</idno><idno type="doi">10.1007/978-1-61779-095-9_2</idno><idno type="wicri:Area/PubMed/Corpus">000A15</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000A15</idno><idno type="wicri:Area/PubMed/Curation">000975</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">000975</idno><idno type="wicri:Area/PubMed/Checkpoint">000975</idno><idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">000975</idno><idno type="wicri:Area/Ncbi/Merge">000D19</idno><idno type="wicri:Area/Ncbi/Curation">000D19</idno><idno type="wicri:Area/Ncbi/Checkpoint">000D19</idno><idno type="wicri:Area/Main/Merge">001644</idno><idno type="wicri:Area/Main/Curation">001564</idno><idno type="wicri:Area/Main/Exploration">001564</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Introduction to gene therapy: a clinical aftermath.</title><author><name sortKey="Denefle, Patrice P" sort="Denefle, Patrice P" uniqKey="Denefle P" first="Patrice P" last="Denèfle">Patrice P. Denèfle</name><affiliation wicri:level="3"><nlm:affiliation>Translational Sciences, IPSEN, and Biotherapies, ParisTech Institute, Paris-Descartes University, Paris, France. pdenefle@gmail.com</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Translational Sciences, IPSEN, and Biotherapies, ParisTech Institute, Paris-Descartes University, Paris</wicri:regionArea><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author></analytic><series><title level="j">Methods in molecular biology (Clifton, N.J.)</title><idno type="eISSN">1940-6029</idno><imprint><date when="2011" type="published">2011</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Animals</term><term>Clinical Trials as Topic</term><term>Cystic Fibrosis (genetics)</term><term>Cystic Fibrosis (therapy)</term><term>Genetic Therapy (legislation & jurisprudence)</term><term>Genetic Therapy (methods)</term><term>Genetic Therapy (trends)</term><term>Genetic Vectors (therapeutic use)</term><term>Humans</term><term>Lipoprotein Lipase (genetics)</term><term>Lipoprotein Lipase (therapeutic use)</term><term>Muscular Dystrophy, Duchenne (genetics)</term><term>Muscular Dystrophy, Duchenne (therapy)</term><term>Parkinson Disease (genetics)</term><term>Parkinson Disease (therapy)</term><term>Peripheral Vascular Diseases (genetics)</term><term>Peripheral Vascular Diseases (therapy)</term><term>Severe Combined Immunodeficiency (therapy)</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Lipoprotein Lipase</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Cystic Fibrosis</term><term>Muscular Dystrophy, Duchenne</term><term>Parkinson Disease</term><term>Peripheral Vascular Diseases</term></keywords><keywords scheme="MESH" qualifier="legislation & jurisprudence" xml:lang="en"><term>Genetic Therapy</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Genetic Therapy</term></keywords><keywords scheme="MESH" qualifier="therapeutic use" xml:lang="en"><term>Genetic Vectors</term><term>Lipoprotein Lipase</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Cystic Fibrosis</term><term>Muscular Dystrophy, Duchenne</term><term>Parkinson Disease</term><term>Peripheral Vascular Diseases</term><term>Severe Combined Immunodeficiency</term></keywords><keywords scheme="MESH" qualifier="trends" xml:lang="en"><term>Genetic Therapy</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Animals</term><term>Clinical Trials as Topic</term><term>Humans</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Despite three decades of huge progress in molecular genetics, in cloning of disease causative gene as well as technology breakthroughs in viral biotechnology, out of thousands of gene therapy clinical trials that have been initiated, only very few are now reaching regulatory approval. We shall review some of the major hurdles, and based on the current either positive or negative examples, we try to initiate drawing a learning curve from experience and possibly identify the major drivers for future successful achievement of human gene therapy trials.</div></front></TEI><affiliations><list><country><li>France</li></country><region><li>Île-de-France</li></region><settlement><li>Paris</li></settlement></list><tree><country name="France"><region name="Île-de-France"><name sortKey="Denefle, Patrice P" sort="Denefle, Patrice P" uniqKey="Denefle P" first="Patrice P" last="Denèfle">Patrice P. Denèfle</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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