Evidence map›Paper›PMID 19104914›Full record

ArticlePharmaceutical research2009

Near complete rescue of experimental Parkinson's disease with intravenous, non-viral GDNF gene therapy.

Yun Zhang, William M Pardridge

Open access · hybridAbstract read
PubMed Publisher
In one paragraph

Article in Pharmaceutical research, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 1 pooled it
1.4field-weighted citation impact, top 22% 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

24 citing papers in PubMed, 1 synthesis or guideline pooled it, 77 citations in OpenAlex.

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  18. Systemic exosomal siRNA delivery reduced alpha-synuclein aggregates in brains of transgenic mice.Movement disorders : official journal of the Movement Disorder Society · 2014
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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

2 authors at 1 institution in 1 country.

Yun ZhangDepartment of Medicine, UCLA, Warren Hall 13-164, 900 Veteran Ave., Los Angeles, California 90024, USA.
William M Pardridge
UCLA Health · US

Funding

Non-Viral Gene Targeting to the BrainR01NS053540 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI PARDRIDGE, WILLIAM M · 2006 to 2008
$920k
NINDS NIH HHS NS-53540
6 · The paper itself

Abstract

purposeRats with experimental Parkinson's disease (PD) are treated with intravenous glial-derived neurotrophic factor (GDNF) plasmid DNA and non-viral gene therapy using Trojan horse liposomes (THLs) targeted with a monoclonal antibody (MAb) to the rat transferrin receptor (TfR). The GDNF transgene expression is under the influence of the rat tyrosine hydroxylase (TH) promoter.

methodsThe GDNF expression plasmid is designated pTHproGDNF. Rats were treated with 3 weekly injections of THLs starting 1 week after the intra-cerebral injection of 6-hydroxydopamine. The dose of the pTHproGDNF was 10 mICROg/rat/weekly injection. Rats were tested with three assays of neurobehavior, and terminal striatal TH enzyme activity was measured at 6 weeks following toxin administration, which is 3 weeks following the last administration of THLs.

resultsApomorphine-induced contralateral rotation was reduced 87% by THL gene therapy; amphetamine-induced ipsilateral rotation was reduced 90% by THL gene therapy; whisker-induced forelimb placement abnormalities were reduced 77% with THL gene therapy. The improvement in neurobehavior correlated with a lasting 77% increase in striatal TH enzyme activity, relative to saline treated rats.

conclusionsNear complete abrogation of the neurotoxin effects are achieved with multiple intravenous dosing of GDNF plasmid DNA gene therapy, using receptor-targeted THLs, and a region-specific promoter.

Indexed as

Genetic TherapyAnimalsAntibodies, MonoclonalGlial Cell Line-Derived Neurotrophic FactorLiposomesMaleParkinsonian DisordersPlasmidsRatsRats, Sprague-DawleyReceptors, TransferrinTyrosine 3-MonooxygenaseAntibodies, MonoclonalGlial Cell Line-Derived Neurotrophic FactorLiposomesReceptors, TransferrinTyrosine 3-Monooxygenase

Identifiers

PMID19104914
OpenAlexW2003918355

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.