[Gene therapy in Parkinson disease: a promising future treatment?].
Identifieur interne : 000911 ( PubMed/Curation ); précédent : 000910; suivant : 000912[Gene therapy in Parkinson disease: a promising future treatment?].
Auteurs : Claire Golé [France] ; Romain Reboul ; Pascale PisanoSource :
- Therapie [ 0040-5957 ]
English descriptors
- KwdEn :
- MESH :
- chemical , genetics : Aromatic-L-Amino-Acid Decarboxylases, Glutamate Decarboxylase, Tyrosine 3-Monooxygenase.
- methods : Genetic Therapy.
- therapy : Parkinson Disease.
- trends : Genetic Therapy.
- Clinical Trials as Topic, Genetic Vectors, Humans.
Abstract
Parkinson disease is a neurodegenerative pathology with high incidence. Current treatments ease the symptoms but don't stop the development of the disease and aren't without any major side effects. Although this pathology is not specifically caused by genetic abnormalities, the involvement of numerous proteins in the pathophysiological process enables us to give an interest to gene therapy. This hypothesis has been upheld by positive results on animals and by five phase I trials on humans but may be attenuated by the first phase II trial recently published showing modest efficiency and multiple side effects. However, these preliminary results will need to be reinforced by more important trials in order to be sure of the safety and get efficacy data, which will allow us to give an opinion upon this new way of treatment.
DOI: 10.2515/therapie/2011062
PubMed: 22031685
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pubmed:22031685Le document en format XML
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<author><name sortKey="Gole, Claire" sort="Gole, Claire" uniqKey="Gole C" first="Claire" last="Golé">Claire Golé</name>
<affiliation wicri:level="1"><nlm:affiliation>Hopital de la Timone, Marseille, France. claire.gole@ap-hm.fr</nlm:affiliation>
<country xml:lang="fr">France</country>
<wicri:regionArea>Hopital de la Timone, Marseille</wicri:regionArea>
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<author><name sortKey="Reboul, Romain" sort="Reboul, Romain" uniqKey="Reboul R" first="Romain" last="Reboul">Romain Reboul</name>
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<author><name sortKey="Pisano, Pascale" sort="Pisano, Pascale" uniqKey="Pisano P" first="Pascale" last="Pisano">Pascale Pisano</name>
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<term>Genetic Therapy (methods)</term>
<term>Genetic Therapy (trends)</term>
<term>Genetic Vectors</term>
<term>Glutamate Decarboxylase (genetics)</term>
<term>Humans</term>
<term>Parkinson Disease (therapy)</term>
<term>Tyrosine 3-Monooxygenase (genetics)</term>
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<keywords scheme="MESH" qualifier="trends" xml:lang="en"><term>Genetic Therapy</term>
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<front><div type="abstract" xml:lang="en">Parkinson disease is a neurodegenerative pathology with high incidence. Current treatments ease the symptoms but don't stop the development of the disease and aren't without any major side effects. Although this pathology is not specifically caused by genetic abnormalities, the involvement of numerous proteins in the pathophysiological process enables us to give an interest to gene therapy. This hypothesis has been upheld by positive results on animals and by five phase I trials on humans but may be attenuated by the first phase II trial recently published showing modest efficiency and multiple side effects. However, these preliminary results will need to be reinforced by more important trials in order to be sure of the safety and get efficacy data, which will allow us to give an opinion upon this new way of treatment.</div>
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<Abstract><AbstractText>Parkinson disease is a neurodegenerative pathology with high incidence. Current treatments ease the symptoms but don't stop the development of the disease and aren't without any major side effects. Although this pathology is not specifically caused by genetic abnormalities, the involvement of numerous proteins in the pathophysiological process enables us to give an interest to gene therapy. This hypothesis has been upheld by positive results on animals and by five phase I trials on humans but may be attenuated by the first phase II trial recently published showing modest efficiency and multiple side effects. However, these preliminary results will need to be reinforced by more important trials in order to be sure of the safety and get efficacy data, which will allow us to give an opinion upon this new way of treatment.</AbstractText>
<CopyrightInformation>© 2011 Société Française de Pharmacologie et de Thérapeutique.</CopyrightInformation>
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<VernacularTitle>La thérapie génique dans la maladie de Parkinson : un traitement d'avenir ?</VernacularTitle>
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