Evidence map›Paper›PMID 15717064›Full record

ReviewNeuroRx : the journal of the American Society for Experimental NeuroTherapeutics2005

Tyrosine hydroxylase replacement in experimental Parkinson's disease with transvascular gene therapy.

William M Pardridge

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

Review in NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
1.4field-weighted citation impact, top 21% 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

25 citing papers in PubMed, 98 citations in OpenAlex.

  1. Review
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  4. Review
  5. Current Chemical, Biological, and Physiological Views in the Development of Successful Brain-Targeted Pharmaceutics.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022
    Review
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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

1 author at 1 institution in 1 country.

William M PardridgeDepartment of Medicine, UCLA, Los Angeles, California 90024, USA. wpardridge@mednet.ucla.edu
UCLA Health · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Transvascular gene therapy of Parkinson's disease (PD) is a new approach to the gene therapy of PD and involves the global distribution of a therapeutic gene to brain after an intravenous administration and transport across the blood-brain barrier (BBB). This is enabled with the development of a nonviral gene transfer technology that encapsulates plasmid DNA inside pegylated immunoliposomes or PILs. An 85- to 100-nm liposome carries the DNA inside the nanocontainer, and the liposome surface is conjugated with several thousand strands of 2000-Da polyethyleneglycol (PEG). This PEGylation of the liposome stabilizes the structure in the blood stream. The liposome is targeted across the BBB via attachment to the tips of 1-2% of the PEG strands of a receptor-specific monoclonal antibody (mAb) directed at a BBB receptor, such as the insulin receptor or transferrin receptor (TfR). Owing to the expression of the insulin receptor or the TfR on both the BBB and the neuronal plasma membrane, the PIL is able to reach the neuronal nuclear compartment from the circulation. Brain-specific expression is possible with the combined use of the PIL gene transfer technology and brain-specific gene promoters. In the 6-hydroxydopamine rat model of experimental PD, striatal tyrosine hydroxylase (TH) activity is completely normalized after an intravenous administration of TfRmAb-targeted PILs carrying a TH expression plasmid. A treatment for PD may be possible with dual gene therapy that seeks both to replace striatal TH gene expression with TH gene therapy, and to halt or reverse neurodegeneration of the nigro-striatal tract with neurotrophin gene therapy.

Indexed as

Genetic TherapyAnimalsBlood-Brain BarrierHumansLiposomesParkinson Disease, SecondaryPolyethylene GlycolsTyrosine 3-MonooxygenaseLiposomesPolyethylene GlycolsTyrosine 3-Monooxygenase

Identifiers

PMID15717064
PMCPMC539333
OpenAlexW1966097940

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.