Biology Reference
In-Depth Information
Chapter 17
Viral Vectors in Primate Research:
Examples from Parkinson's Disease Research
Grégory Porras , Benjamin Dehay , and Erwan Bezard
Abstract
Neurodegenerative diseases, such as Parkinson's disease (PD), offer unique opportunities for the therapeu-
tic application of viral vector gene transfer. In this chapter, we discuss the current state of viral vector
preclinical development in nonhuman primate with special focus on vectors derived from adeno-associated
virus (AAV) and lentivirus (LV), as they are the only ones that have reached clinical trials. We also outline
desired features for improving the routes of delivery, tropism specifi city, and other parameters that infl u-
ence achievement of successful therapeutic goals.
Key words
Nonhuman primate, Central nervous system, Viral vectors, Parkinson's disease, Gene
therapies, Gene transfer
Abbreviations
6-OHDA 6-Hydroxydopamine
AADC
Aromatic
L
-amino acid decarboxylase
AAV
Adeno-associated virus
CNS
Central nervous system
D1
Dopamine D1 receptors
D2
Dopamine D2 receptors
GABA
γ
-Aminobutyric acid
GAD
Glutamic acid decarboxylase
GCH
Guanosine triphosphate cyclohydrolase I
GDNF
Glial cell line-derived neurotrophic factor
GRKs
G protein coupled receptor
GTPase
Guanosine triphosphatase
L
-DOPA
L
-3,4-Dihydroxyphenylalanine
LID
L
-DOPA-induced dyskinesia
LV
Lentivirus
MPTP
1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine
MRI
Magnetic resonance image
NPH
Nonhuman primate
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