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Herzog, C.D., Dass, B., Holden, J.E., Stansell, J. 3rd, Gasmi, M., Tuszynski, M.H., Bartus, R.T. and Kordower, J.H. (2007) Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys. Movement Disorders, 22, 1124-1132.
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TITLE:
Can population genomics guide future therapeutic gene transfer strategies for Parkinson’s disease?
AUTHORS:
Massimo S. Fiandaca, Robert M. Padilla, Ishmeal Conteh, Howard J. Federoff
KEYWORDS:
Biomarkers; Functional Imaging; Gene Therapy; Parkinson’s Disease; Population Genomics; Prodromal Phase
JOURNAL NAME:
Open Journal of Genetics,
Vol.3 No.2A,
June
26,
2013
ABSTRACT: Medical and surgical therapies for patients with Parkinson’s disease (PD)
are typically considered and initiated upon development of clinical signs, especially
therapeutic gene transfer therapies. Early clinical trials delivering transgenes
within the brains of PD patients have confirmed their safety and suggested mild
to moderate efficacy. Confirmatory phase III trials have yet to be undertaken
with any of the current treatment regimens. During the development of PD gene
therapy, mapping of the human genome was finalized and provides major insights
into the normal and pathogenic genetic variabilities of populations. Genome
wide association studies (GWAS) have expanded the genetic defects and risk
factors accompanying clinical PD. Advanced genomic investigations may allow
asymptomatic individuals with a high risk of developing PD, and evident
presymptomatic nigrostriatal deficiencies, to consider early treatment
approaches. Herein we propose that certain genomically and clinically defined
PD patients may provide unique opportunities for testing neuronotrophic gene
therapy in a pathobiological environment that is antecedent to overt motoric
dysfunction. Such an approach may finally allow testing of the disease-altering
capabilities of therapeutic gene transfer in PD.