Evidence map›Paper›PMID 25205986›Full record

ReviewAnnals of neurosciences2012

Gene-based therapy of Parkinson's Disease: Translation from animal model to human clinical trial employing convection enhanced delivery.

Gurwattan S Miranpuri, Lauren Kumbier, Angelica Hinchman, Dominic Schomberg, Anyi Wang, Hope Marshall, Ken Kubota, Chris Ross, Karl Sillay

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Annals of neurosciences, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.6field-weighted citation impact, top 30% 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

8 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Review
  3. [Effect of convection enhanced delivery on the microstructure of brain extracellular space in aged rats].Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health sciences · 2020
    Article
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
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

9 authors at 2 institutions in 1 country.

Gurwattan S MiranpuriDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Lauren KumbierDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Angelica HinchmanDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Dominic SchombergDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Anyi WangDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Hope MarshallDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Ken KubotaKinetic Foundation, Los Altos, CA, 94023, USA;
Chris RossEngineering Resources Group Inc, Pembroke Pines, FL, 33029, USA.
Karl SillayDepartment of Neurological Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA;
Neurological Surgery · USUniversity of Wisconsin–Madison · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The existing treatment of Parkinson's disease (PD) is directed towards substituting dopamine loss with either dopamine replacement therapy or pharmacological therapies aimed at increasing dopamine at the synapse level. Emerging viable alternatives include the use of cell-based and gene-based therapeutics. In this review, we discuss efforts in developing in vitro and in vivo models and their translation to human clinical trials for gene-based therapy of this distressing and prevalent neurodegenerative disorder. Given the mismatch between expectations from preclinical data and results of human pivotal trials, drug delivery has been identified as the key emerging area for translational research due to limitation of limited efficacy. The chief highlights of the current topic include use of improved delivery methods of gene-based therapeutic agents. Convection-enhanced delivery (CED), an advanced infusion technique with demonstrated utility in ex vivo and in vivo animal models has recently been adopted for PD gene-based therapy trials. Several preclinical studies suggest that magnetic resonance imaging (MRI)-guided navigation for accurately targeting and real time monitoring viral vector delivery (rCED) in future clinical trials involving detection of gene expression and restoration of dopaminergic function loss using pro-drug approach will greatly enhance these PD treatments.

Indexed as

Gene-based therapyParkinson’s diseaseVector

Identifiers

PMID25205986
PMCPMC4117084
OpenAlexW2008404899

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.