Evidence map›Paper›PMID 35957524›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2022

Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson's disease.

Matthew T Rocco, Asad S Akhter, Debra J Ehrlich, Gretchen C Scott, Codrin Lungu, Vikas Munjal, Anthony Aquino, Russell R Lonser, Massimo S Fiandaca, Mark Hallett and 2 more

Erratum issuedOpen access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 30 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 2 pooled it
6.3field-weighted citation impact, top 3% 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

30 citing papers in PubMed, 2 syntheses or guidelines pooled it, 53 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Review
  5. Article
  6. Article
  7. A Phase 1 Study of Convection-Enhanced Delivery of Intraputaminal AAV2-GDNF in Advanced Parkinson's Disease.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  8. Article
  9. Clinical GDNF delivery methods for Parkinson's disease.Journal of Parkinson's disease · 2026
    Review
  10. Genetic therapies for neurological diseases.Pharmacological reviews · 2026
    Review
  11. Article
  12. Article
  13. Review
  14. Article
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  17. Article
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 5 institutions in 1 country.

Matthew T RoccoDepartment of Neurological Surgery, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Asad S AkhterDepartment of Neurological Surgery, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Debra J EhrlichParkinson's Disease Clinic, NINDS, National Institutes of Health Division of Clinical Research, Bethesda, MD 20896, USA.
Gretchen C ScottSurgical Neurology Branch, NINDS, National Institutes of Health, Bethesda, MD 20896, USA.
Codrin LunguDivision of Clinical Research, NINDS, National Institutes of Health, Bethesda, MD 20896, USA.
Vikas MunjalDepartment of Neurological Surgery, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Anthony AquinoDepartment of Radiology, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Russell R LonserDepartment of Neurological Surgery, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
Massimo S FiandacaAsklepios BioPharmaceutical, Inc., 2447 North Star Road, Upper Arlington, OH 43221, USA.
Mark HallettDivision of Clinical Research, NINDS, National Institutes of Health, Bethesda, MD 20896, USA; Human Motor Control Section, Medical Neurology Branch, NINDS, National Institutes of Health, Bethesda, MD 20896, USA.
John D HeissSurgical Neurology Branch, NINDS, National Institutes of Health, Bethesda, MD 20896, USA.
Krystof S BankiewiczDepartment of Neurological Surgery, College of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA. Electronic address: krzysztof.bankiewicz@osumc.edu.
The Ohio State University Wexner Medical Center · USNational Institutes of Health · USNational Institute of Neurological Disorders and Stroke · USNeurological Surgery · USAskBio (United States) · US

Funding

Central Nervous System Drug Delivery TechniquesZIANS003051 · NINDS · NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE · PI HEISS, JOHN · 2009 to 2025
$9.8M
6 · The paper itself

Abstract

Direct putaminal infusion of adeno-associated virus vector (serotype 2) (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene was studied in a phase I clinical trial of participants with advanced Parkinson's disease (PD). Convection-enhanced delivery of AAV2-GDNF with a surrogate imaging tracer (gadoteridol) was used to track infusate distribution during real-time intraoperative magnetic resonance imaging (iMRI). Pre-, intra-, and serial postoperative (up to 5 years after infusion) MRI were analyzed in 13 participants with PD treated with bilateral putaminal co-infusions (52 infusions in total) of AAV2-GDNF and gadoteridol (infusion volume, 450 mL per putamen). Real-time iMRI confirmed infusion cannula placement, anatomic quantification of volumetric perfusion within the putamen, and direct visualization of off-target leakage or cannula reflux (which permitted corresponding infusion rate/cannula adjustments). Serial post-treatment MRI assessment (n = 13) demonstrated no evidence of cerebral parenchyma toxicity in the corresponding regions of AAV2-GDNF and gadoteridol co-infusion or surrounding regions over long-term follow-up. Direct confirmation of key intraoperative safety and efficacy parameters underscores the safety and tissue targeting value of real-time imaging with co-infused gadoteridol and putative therapeutic agents (i.e., AAV2-GDNF). This delivery-imaging platform enhances safety, permits delivery personalization, improves therapeutic distribution, and facilitates assessment of efficacy and dosing effect.

Indexed as

Parkinson DiseaseHumansMagnetic Resonance ImagingAAVconvection-enhanced deliverygadoteridolGDNFgene therapyMRI-guidedParkinson's diseaseputamensafety

Identifiers

PMID35957524
PMCPMC9734022
OpenAlexW4292414316

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.