Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
Identifieur interne : 000099 ( PascalFrancis/Corpus ); précédent : 000098; suivant : 000100Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
Auteurs : Raymond T. Bartus ; Tiffany L. Baumann ; Joao Siffert ; Christopher D. Herzog ; Ron Alterman ; Nicholas Boulis ; Dennis A. Turner ; Mark Stacy ; Anthony E. Lang ; Andres M. Lozano ; C. Warren OlanowSource :
- Neurology [ 0028-3878 ] ; 2013.
Descripteurs français
- Pascal (Inist)
English descriptors
- KwdEn :
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.
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Pour connaître la documentation sur le format Inist Standard.
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Format Inist (serveur)
NO : | PASCAL 13-0179282 INIST |
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ET : | Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients |
AU : | BARTUS (Raymond T.); BAUMANN (Tiffany L.); SIFFERT (Joao); HERZOG (Christopher D.); ALTERMAN (Ron); BOULIS (Nicholas); TURNER (Dennis A.); STACY (Mark); LANG (Anthony E.); LOZANO (Andres M.); WARREN OLANOW (C.) |
AF : | Ceregene, Inc./San Diego, CA/Etats-Unis (1 aut., 2 aut., 3 aut., 4 aut.); Mount Sinai School of Medicine/New York, NY/Etats-Unis (5 aut., 11 aut.); Emory University/Atlanta, GA/Etats-Unis (6 aut.); Duke University/Durham, NC/Etats-Unis (7 aut., 8 aut.); University of Toronto/Toronto/Canada (9 aut., 10 aut.); The Edmond J. Safra Program in Parkinson's Disease Toronto Western Hospital/Toronto/Canada (9 aut.); Avanir Pharmaceuticals/Aliso Viejo, CA/Etats-Unis (3 aut.); Beth Israel Deaconess Medical Center/Boston, MA/Etats-Unis (5 aut.) |
DT : | Publication en série; Niveau analytique |
SO : | Neurology; ISSN 0028-3878; Coden NEURAI; Etats-Unis; Da. 2013; Vol. 80; No. 18; Pp. 1698-1701; Bibl. 13 ref. |
LA : | Anglais |
EA : | 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. |
CC : | 002B17; 002B17G |
FD : | Pathologie du système nerveux; Sécurité; Faisabilité; Ciblage; Locus niger; Homme |
FG : | Encéphale; Système nerveux central |
ED : | Nervous system diseases; Safety; Feasibility; Targeting; Locus niger; Human |
EG : | Encephalon; Central nervous system |
SD : | Sistema nervioso patología; Seguridad; Practicabilidad; Blancado; Locus níger; Hombre |
LO : | INIST-6345.354000504129080140 |
ID : | 13-0179282 |
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Pascal:13-0179282Le document en format XML
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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</title>
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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>
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<term>Targeting</term>
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<keywords scheme="Pascal" xml:lang="fr"><term>Pathologie du système nerveux</term>
<term>Sécurité</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>
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<fC01 i1="01" l="ENG"><s0>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.</s0>
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<s5>01</s5>
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<s5>01</s5>
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<s5>01</s5>
</fC03>
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<s5>09</s5>
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<s5>09</s5>
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<s5>09</s5>
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<s5>10</s5>
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<s5>10</s5>
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<s5>10</s5>
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<s5>11</s5>
</fC03>
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<s5>11</s5>
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<s5>11</s5>
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<s5>12</s5>
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<s5>12</s5>
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<s5>12</s5>
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<s5>13</s5>
</fC03>
<fC03 i1="06" i2="X" l="ENG"><s0>Human</s0>
<s5>13</s5>
</fC03>
<fC03 i1="06" i2="X" l="SPA"><s0>Hombre</s0>
<s5>13</s5>
</fC03>
<fC07 i1="01" i2="X" l="FRE"><s0>Encéphale</s0>
<s5>37</s5>
</fC07>
<fC07 i1="01" i2="X" l="ENG"><s0>Encephalon</s0>
<s5>37</s5>
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<fC07 i1="01" i2="X" l="SPA"><s0>Encéfalo</s0>
<s5>37</s5>
</fC07>
<fC07 i1="02" i2="X" l="FRE"><s0>Système nerveux central</s0>
<s5>38</s5>
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<s5>38</s5>
</fC07>
<fC07 i1="02" i2="X" l="SPA"><s0>Sistema nervioso central</s0>
<s5>38</s5>
</fC07>
<fN21><s1>161</s1>
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<fN44 i1="01"><s1>OTO</s1>
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<fN82><s1>OTO</s1>
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<server><NO>PASCAL 13-0179282 INIST</NO>
<ET>Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</ET>
<AU>BARTUS (Raymond T.); BAUMANN (Tiffany L.); SIFFERT (Joao); HERZOG (Christopher D.); ALTERMAN (Ron); BOULIS (Nicholas); TURNER (Dennis A.); STACY (Mark); LANG (Anthony E.); LOZANO (Andres M.); WARREN OLANOW (C.)</AU>
<AF>Ceregene, Inc./San Diego, CA/Etats-Unis (1 aut., 2 aut., 3 aut., 4 aut.); Mount Sinai School of Medicine/New York, NY/Etats-Unis (5 aut., 11 aut.); Emory University/Atlanta, GA/Etats-Unis (6 aut.); Duke University/Durham, NC/Etats-Unis (7 aut., 8 aut.); University of Toronto/Toronto/Canada (9 aut., 10 aut.); The Edmond J. Safra Program in Parkinson's Disease Toronto Western Hospital/Toronto/Canada (9 aut.); Avanir Pharmaceuticals/Aliso Viejo, CA/Etats-Unis (3 aut.); Beth Israel Deaconess Medical Center/Boston, MA/Etats-Unis (5 aut.)</AF>
<DT>Publication en série; Niveau analytique</DT>
<SO>Neurology; ISSN 0028-3878; Coden NEURAI; Etats-Unis; Da. 2013; Vol. 80; No. 18; Pp. 1698-1701; Bibl. 13 ref.</SO>
<LA>Anglais</LA>
<EA>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.</EA>
<CC>002B17; 002B17G</CC>
<FD>Pathologie du système nerveux; Sécurité; Faisabilité; Ciblage; Locus niger; Homme</FD>
<FG>Encéphale; Système nerveux central</FG>
<ED>Nervous system diseases; Safety; Feasibility; Targeting; Locus niger; Human</ED>
<EG>Encephalon; Central nervous system</EG>
<SD>Sistema nervioso patología; Seguridad; Practicabilidad; Blancado; Locus níger; Hombre</SD>
<LO>INIST-6345.354000504129080140</LO>
<ID>13-0179282</ID>
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