Evidence map›Paper›PMID 32732384›Full record

ReviewJournal of neurology, neurosurgery, and psychiatry2020

Data-driven evolution of neurosurgical gene therapy delivery in Parkinson's disease.

R Mark Richardson, Krystof S Bankiewicz, Chadwick W Christine, Amber D Van Laar, Robert E Gross, Russell Lonser, Stewart A Factor, Sandra K Kostyk, Adrian P Kells, Bernard Ravina and 1 more

Registry-linked trialOpen access · hybridAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Journal of neurology, neurosurgery, and psychiatry, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03733496 (An Observational, Long-Term Extension Study for Participants in Prior VY-AADC01 Clinical Studies), which is not on this map. Cited by 31 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 2 pooled it
1.3field-weighted citation impact, top 16% 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.

NCT03733496 completednot on this map

An Observational, Long-Term Extension Study for Participants in Prior VY-AADC01 Clinical Studies

TypeobservationalSponsorNeurocrine BiosciencesRan2018 to 2023Enrolled14ConditionsParkinson's Disease
3 · Its place in the literature

Who cites it

31 citing papers in PubMed, 2 syntheses or guidelines pooled it, 41 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Review
  4. Clinical GDNF delivery methods for Parkinson's disease.Journal of Parkinson's disease · 2026
    Review
  5. Review
  6. Article
  7. Review
  8. Review
  9. Article
  10. Review
  11. Review
  12. Article
  13. Review
  14. Review
  15. Advancements in surgical treatments for Huntington disease: From pallidotomy to experimental therapies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  16. Review
  17. Neurosurgical gene therapy for central nervous system diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  18. Article
  19. Review
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 7 institutions in 1 country.

R Mark RichardsonDepartment of Neurosurgery, Massachusetts General Hospital, Boston, Massachusetts, USA mark.richardson@mgh.harvard.edu.ORCID 0000-0003-2620-7387
Krystof S BankiewiczDepartment of Neurological Surgery, University of California San Francisco, San Francisco, California, USA.
Chadwick W ChristineDepartment of Neurology, University of California San Francisco, San Francisco, California, USA.
Amber D Van LaarDepartment of Neurology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA.
Robert E GrossDepartment of Neurosurgery, Emory University, Atlanta, Georgia, USA.
Russell LonserDepartment of Neurological Surgery, Ohio State University College of Medicine, Columbus, Ohio, USA.
Stewart A FactorDepartment of Neurology, Emory University, Atlanta, Georgia, USA.
Sandra K KostykDepartments of Neuroscience and Neurology, Ohio State University College of Medicine, Columbus, Ohio, USA.
Adrian P KellsBrain Neurotherapy Bio, Inc, Columbus, Ohio, USA.
Bernard RavinaPraxis Precision Medicines, Inc, Cambridge, Massachusetts, USA.
Paul S LarsonDepartment of Neurological Surgery, University of California San Francisco, San Francisco, California, USA.
University of California, San Francisco · USEmory University · USHarvard University · USNeurological Surgery · USPraxis (United States) · USThe Ohio State University · USUniversity of Pittsburgh Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Loss of nigrostriatal dopaminergic projection neurons is a key pathology in Parkinson's disease, leading to abnormal function of basal ganglia motor circuits and the accompanying characteristic motor features. A number of intraparenchymally delivered gene therapies designed to modify underlying disease and/or improve clinical symptoms have shown promise in preclinical studies and subsequently were evaluated in clinical trials. Here we review the challenges with surgical delivery of gene therapy vectors that limited therapeutic outcomes in these trials, particularly the lack of real-time monitoring of vector administration. These challenges have recently been addressed during the evolution of novel techniques for vector delivery that include the use of intraoperative MRI. The preclinical development of these techniques are described in relation to recent clinical translation in an adeno-associated virus serotype 2-mediated human aromatic L-amino acid decarboxylase gene therapy development programme. This new paradigm allows visualisation of the accuracy and adequacy of viral vector delivery within target structures, enabling intertrial modifications in surgical approaches, cannula design, vector volumes and dosing. The rapid, data-driven evolution of these procedures is unique and has led to improved vector delivery.

Indexed as

Corpus StriatumMagnetic Resonance ImagingSubstantia NigraAnimalsAromatic-L-Amino-Acid DecarboxylasesBasal GangliaDependovirusEvidence-Based MedicineGenetic TherapyGenetic VectorsGlutamate DecarboxylaseGTP CyclohydrolaseHumansIntraoperative CareLentivirusNeurosurgical ProceduresAromatic-L-Amino-Acid DecarboxylasesDDC protein, humanGlutamate DecarboxylaseGTP CyclohydrolaseNeurturinNRTN protein, humanTyrosine 3-MonooxygenaseneurosurgeryParkinson's disease

Identifiers

PMID32732384
PMCPMC7569395
OpenAlexW3046672751

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

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.