ParkinsonCanadaV1, Main, Exploration, bibRecord, 001047

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Identifieur interne : 001047 ( Main/Exploration ); précédent : 001046; suivant : 001048

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Auteurs : Raymond T. Bartus [États-Unis] ; Tiffany L. Baumann [États-Unis] ; Joao Siffert [États-Unis] ; Christopher D. Herzog [États-Unis] ; Ron Alterman [États-Unis] ; Nicholas Boulis [États-Unis] ; Dennis A. Turner [États-Unis] ; Mark Stacy [États-Unis] ; Anthony E. Lang [Canada] ; Andres M. Lozano [Canada] ; C. Warren Olanow [États-Unis]

Source :

Neurology [ 0028-3878 ] ; 2013.

RBID : Pascal:13-0179282

Descripteurs français

Pascal (Inist)
- Pathologie du système nerveux, Sécurité, Faisabilité, Ciblage, Locus niger, Homme.
Wicri :
- topic : Homme.

English descriptors

KwdEn :
- Feasibility, Human, Locus niger, Nervous system diseases, Safety, Targeting.

Abstract

Objective: In an effort to account for deficiencies in axonal transport that limit the effectiveness of neurotrophic factors, this study tested the safety and feasibility, in moderately advanced Parkinson disease (PD), of bilaterally administering the gene therapy vector AAV2-neurturin (CERE-120) to the putamen plus substantia nigra (SN, a relatively small structure deep within the midbrain, in proximity to critical neuronal and vascular structures). Methods: After planning and minimizing risks of stereotactically targeting the SN, an open-label, dose-escalation safety trial was initiated in 6 subjects with PD who received bilateral stereotactic injections of CERE-120 into the SN and putamen. Results: Two-year safety data for all subjects suggest the procedures were well-tolerated, with no serious adverse events. All adverse events and complications were expected for patients with PD undergoing stereotactic brain surgery. Conclusions: Bilateral stereotactic administration of CERE-120 to the SN plus putamen in PD is feasible and this evaluation provides initial empirical support that it is safe and well-tolerated. Classification of evidence: This study provides Class IV evidence that bilateral neurturin gene delivery (CERE-120) to the SN plus putamen in patients with moderately advanced PD is feasible and safe.

Url:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3716474

Affiliations:

Le document en format XML

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<author><name sortKey="Warren Olanow, C" sort="Warren Olanow, C" uniqKey="Warren Olanow C" first="C." last="Warren Olanow">C. Warren Olanow</name>
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<series><title level="j" type="main">Neurology</title>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Feasibility</term>
<term>Human</term>
<term>Locus niger</term>
<term>Nervous system diseases</term>
<term>Safety</term>
<term>Targeting</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Pathologie du   système nerveux</term>
<term>Sécurité</term>
<term>Faisabilité</term>
<term>Ciblage</term>
<term>Locus niger</term>
<term>Homme</term>
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<front><div type="abstract" xml:lang="en">Objective: In an effort to account for deficiencies in axonal transport that limit the effectiveness of neurotrophic factors, this study tested the safety and feasibility, in moderately advanced Parkinson disease (PD), of bilaterally administering the gene therapy vector AAV2-neurturin (CERE-120) to the putamen plus substantia nigra (SN, a relatively small structure deep within the midbrain, in proximity to critical neuronal and vascular structures). Methods: After planning and minimizing risks of stereotactically targeting the SN, an open-label, dose-escalation safety trial was initiated in 6 subjects with PD who received bilateral stereotactic injections of CERE-120 into the SN and putamen. Results: Two-year safety data for all subjects suggest the procedures were well-tolerated, with no serious adverse events. All adverse events and complications were expected for patients with PD undergoing stereotactic brain surgery. Conclusions: Bilateral stereotactic administration of CERE-120 to the SN plus putamen in PD is feasible and this evaluation provides initial empirical support that it is safe and well-tolerated. Classification of evidence: This study provides Class IV evidence that bilateral neurturin gene delivery (CERE-120) to the SN plus putamen in patients with moderately advanced PD is feasible and safe.</div>
</front>
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<name sortKey="Baumann, Tiffany L" sort="Baumann, Tiffany L" uniqKey="Baumann T" first="Tiffany L." last="Baumann">Tiffany L. Baumann</name>
<name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name>
<name sortKey="Herzog, Christopher D" sort="Herzog, Christopher D" uniqKey="Herzog C" first="Christopher D." last="Herzog">Christopher D. Herzog</name>
<name sortKey="Siffert, Joao" sort="Siffert, Joao" uniqKey="Siffert J" first="Joao" last="Siffert">Joao Siffert</name>
<name sortKey="Siffert, Joao" sort="Siffert, Joao" uniqKey="Siffert J" first="Joao" last="Siffert">Joao Siffert</name>
<name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name>
<name sortKey="Turner, Dennis A" sort="Turner, Dennis A" uniqKey="Turner D" first="Dennis A." last="Turner">Dennis A. Turner</name>
<name sortKey="Warren Olanow, C" sort="Warren Olanow, C" uniqKey="Warren Olanow C" first="C." last="Warren Olanow">C. Warren Olanow</name>
</country>
<country name="Canada"><region name="Ontario"><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E." last="Lang">Anthony E. Lang</name>
</region>
<name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E." last="Lang">Anthony E. Lang</name>
<name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M." last="Lozano">Andres M. Lozano</name>
</country>
</tree>
</affiliations>
</record>

Pour manipuler ce document sous Unix (Dilib)

EXPLOR_STEP=$WICRI_ROOT/Wicri/Canada/explor/ParkinsonCanadaV1/Data/Main/Exploration

HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 001047 | SxmlIndent | more

HfdSelect -h $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd -nk 001047 | SxmlIndent | more

Pour mettre un lien sur cette page dans le réseau Wicri

{{Explor lien
   |wiki=    Wicri/Canada
   |area=    ParkinsonCanadaV1
   |flux=    Main
   |étape=   Exploration
   |type=    RBID
   |clé=     Pascal:13-0179282
   |texte=   Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
}}

This area was generated with Dilib version V0.6.29.
Data generation: Thu May 4 22:20:19 2017. Site generation: Fri Dec 23 23:17:26 2022

La maladie de Parkinson au Canada (serveur d'exploration)

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Source :

Descripteurs français

English descriptors

Abstract

Links toward previous steps (curation, corpus...)

Le document en format XML

Pour manipuler ce document sous Unix (Dilib)

Pour mettre un lien sur cette page dans le réseau Wicri

	La maladie de Parkinson au Canada (serveur d'exploration)
	Attention, ce site est en cours de développement ! Attention, site généré par des moyens informatiques à partir de corpus bruts. Les informations ne sont donc pas validées.