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

Publication ,  Journal Article
Bartus, RT; Baumann, TL; Siffert, J; Herzog, CD; Alterman, R; Boulis, N; Turner, DA; Stacy, M; Lang, AE; Lozano, AM; Olanow, CW
Published in: Neurology
April 30, 2013

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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Published In

Neurology

DOI

EISSN

1526-632X

Publication Date

April 30, 2013

Volume

80

Issue

18

Start / End Page

1698 / 1701

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Substantia Nigra
  • Stereotaxic Techniques
  • Parkinson Disease
  • Neurturin
  • Neurology & Neurosurgery
  • Middle Aged
  • Microinjections
  • Male
  • Humans
 

Citation

APA
Chicago
ICMJE
MLA
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Bartus, R. T., Baumann, T. L., Siffert, J., Herzog, C. D., Alterman, R., Boulis, N., … Olanow, C. W. (2013). Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology, 80(18), 1698–1701. https://doi.org/10.1212/WNL.0b013e3182904faa
Bartus, Raymond T., Tiffany L. Baumann, Joao Siffert, Christopher D. Herzog, Ron Alterman, Nicholas Boulis, Dennis A. Turner, et al. “Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients.Neurology 80, no. 18 (April 30, 2013): 1698–1701. https://doi.org/10.1212/WNL.0b013e3182904faa.
Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, et al. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology. 2013 Apr 30;80(18):1698–701.
Bartus, Raymond T., et al. “Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients.Neurology, vol. 80, no. 18, Apr. 2013, pp. 1698–701. Pubmed, doi:10.1212/WNL.0b013e3182904faa.
Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, Turner DA, Stacy M, Lang AE, Lozano AM, Olanow CW. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology. 2013 Apr 30;80(18):1698–1701.

Published In

Neurology

DOI

EISSN

1526-632X

Publication Date

April 30, 2013

Volume

80

Issue

18

Start / End Page

1698 / 1701

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Substantia Nigra
  • Stereotaxic Techniques
  • Parkinson Disease
  • Neurturin
  • Neurology & Neurosurgery
  • Middle Aged
  • Microinjections
  • Male
  • Humans