Combining Gene Transfer and Nonhuman Primates to Better Understand and Treat Parkinson's Disease

Overview

Journal Front Mol Neurosci

Specialty Molecular Biology

Date 2019 Feb 27

PMID 30804750

Citations 7

Authors

Christelle Lasbleiz

Nadine Mestre-Frances

Gina Devau

Maria-Rosario Luquin

Liliane Tenenbaum

Eric J Kremer

Jean-Michel Verdier

Affiliations

Soon will be listed here.

Abstract

Parkinson's disease (PD) is a progressive CNS disorder that is primarily associated with impaired movement. PD develops over decades and is linked to the gradual loss of dopamine delivery to the striatum, the loss of dopaminergic (DA) neurons in the substantia nigra pars compacta (SNpc). While the administration of L-dopa and deep brain stimulation are potent therapies, their costs, side effects and gradual loss of efficacy underlines the need to develop other approaches. Unfortunately, the lack of pertinent animal models that reproduce DA neuron loss and behavior deficits-in a timeline that mimics PD progression-has hindered the identification of alternative therapies. A complementary approach to transgenic animals is the use of nonhuman primates (NHPs) combined with the overexpression of disease-related genes using viral vectors. This approach may induce phenotypes that are not influenced by developmental compensation mechanisms, and that take into account the personality of animals. In this review article, we discuss the combination of gene transfer and NHPs to develop "genetic" models of PD that are suitable for testing therapeutic approaches.

Citing Articles

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gene mutation by pair truncated sgRNA/Cas9-D10A in cynomolgus monkeys.

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Targeted Transgene Expression in Cholinergic Interneurons in the Monkey Striatum Using Canine Adenovirus Serotype 2 Vectors.

Martel A, Elseedy H, Lavigne M, Scapula J, Ghestem A, Kremer E Front Mol Neurosci. 2020; 13:76.

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