Evidence map›Paper›PMID 22424171›Full record

ArticleHuman gene therapy2012

Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease.

Gabriele Mittermeyer, Chadwick W Christine, Kathryn H Rosenbluth, Suzanne L Baker, Philip Starr, Paul Larson, Paul L Kaplan, John Forsayeth, Michael J Aminoff, Krystof S Bankiewicz

Open access · greenAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 141 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
141citing papers in PubMed, 1 pooled it
14.2field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

141 citing papers in PubMed, 1 synthesis or guideline pooled it, 296 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Trial
  4. Trial
  5. Article
  6. Review
  7. Article
  8. Review
  9. Review
  10. Recent developments in translational imaging of in vivo gene therapy outcomes.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  11. Review
  12. Review
  13. Review
  14. From lab bench to hope: a review of gene therapies in clinical trials for Parkinson's disease and challenges.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Review
  15. Review
  16. Review
  17. Role of FoxP3Human gene therapy · 2024
    Review
  18. Article
  19. Review
  20. Review

81 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 1 institution in 1 country.

Gabriele MittermeyerDepartment of Neurological Surgery, University of California San Francisco, San Francisco, CA 94143, USA.
Chadwick W Christine
Kathryn H Rosenbluth
Suzanne L Baker
Philip Starr
Paul Larson
Paul L Kaplan
John Forsayeth
Michael J Aminoff
Krystof S Bankiewicz
University of California, Berkeley · US

Funding

Trophic gene transfer in MPTP animalsU54NS045309 · NINDS · UNIVERSITY OF ROCHESTER · PI FEDEROFF, HOWARD J. · 2002 to 2006
$9.7M
NINDS NIH HHS U54 NS045309
6 · The paper itself

Abstract

We report the results of a long-term follow-up of subjects in a phase 1 study of AAV2-hAADC (adeno-associated virus type 2-human aromatic L-amino acid decarboxylase) gene therapy for the treatment of Parkinson's disease (PD). Ten patients with moderately advanced PD received bilateral putaminal infusions of either a low or a high dose of AAV2-hAADC vector. An annual positron emission tomography (PET) imaging with [(18)F]fluoro-L-m-tyrosine tracer was used for evaluation of AADC expression, and a standard clinical rating scale [Unified Parkinson's Disease Rating Scale (UPDRS)] was used to assess effect. Our previous analysis of the 6-month data suggested that this treatment was acutely safe and well tolerated. We found that the elevated PET signal observed in the first 12 months persisted over 4 years in both dose groups. A significantly increased PET value compared with the presurgery baseline was maintained over the 4-year monitoring period. The UPDRS in all patients off medication for 12 hr improved in the first 12 months, but displayed a slow deterioration in subsequent years. This analysis demonstrates that apparent efficacy continues through later years with an acceptable safety profile. These data indicate stable transgene expression over 4 years after vector delivery and continued safety, but emphasize the need for a controlled efficacy trial and the use of a higher vector dose.

Indexed as

AgedAromatic-L-Amino-Acid DecarboxylasesCohort StudiesDependovirusFemaleGenetic TherapyGenetic VectorsHumansMaleMiddle AgedParkinson DiseasePositron-Emission TomographyAromatic-L-Amino-Acid Decarboxylases

Identifiers

PMID22424171
PMCPMC4530392
OpenAlexW2065259720

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.