ReviewExperimental neurology2008
Regulatable promoters and gene therapy for Parkinson's disease: is the only thing to fear, fear itself?
Review in Experimental neurology, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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.
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.
Who cites it
11 citing papers in PubMed, 32 citations in OpenAlex.
- Adeno-associated Virus-mediated, Mifepristone-regulated Transgene Expression in the Brain.Molecular therapy. Nucleic acids · 2013Article
- A single intramuscular injection of rAAV-mediated mutant erythropoietin protects against MPTP-induced parkinsonism.Genes, brain, and behavior · 2013Article
- Gene therapy for the treatment of Parkinson's disease: the nature of the biologics expands the future indications.Pharmaceuticals (Basel, Switzerland) · 2012Article
- Stem cells and regenerative therapies for Parkinson's disease.Degenerative neurological and neuromuscular disease · 2012Review
- Gene therapy for Parkinson's disease.Parkinson's disease · 2012Article
- Article
- Of monkeys and men: analysis of the phase 2 double-blind, sham-surgery controlled, randomized trial of AAV2-neurturin gene therapy for Parkinson's disease.Current neurology and neuroscience reports · 2011Article
- CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unit.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Tight Long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- alpha-Synuclein: a therapeutic target for Parkinson's disease?Pharmacological researchReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Gene therapy for Parkinson's disease has become a clinical reality with three different approaches currently being tested in patients. All three trials employ an adeno-associated virus with a type two serotype (AAV2). To date, no serious adverse events related to the injections of therapeutic vectors have been reported in any patient. This safety profile was predicted based upon, in some cases, exhaustive preclinical testing in both rodent and primate species. Still some argue that regulatable promoters are required so that expression of the transgene can be halted should untoward side effects arise. We argue that given the current empirical data base of AAV2, the lack of regulatable promoters that have been proven to be safe and effective, and the pressing clinical needs of PD patients, the mandatory use of regulatable vectors is not only unnecessary but, in some instances, misguided and potentially dangerous. This commentary will outline the issues related to the use of regulatable promoters for gene therapy for PD and express our opinion as to why mandating the use of such promoters might result in outcomes that are unsafe, unproductive, and counter to the progress of scientifically sound, clinical research.
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What OpenQuestion holds
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.