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Gene therapy approaches for Parkinson's disease
Author(s) -
Aebischer P.,
Pralong W.
Publication year - 2003
Publication title -
journal of neurochemistry
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.75
H-Index - 229
eISSN - 1471-4159
pISSN - 0022-3042
DOI - 10.1046/j.1471-4159.85.s2.8_3.x
Subject(s) - glial cell line derived neurotrophic factor , neurotrophic factors , substantia nigra , neuroscience , dopaminergic , neuroprotection , striatum , neurturin , parkinson's disease , biology , genetic enhancement , medicine , dopamine , disease , gene , pathology , genetics , receptor
The CNS delivery of glial cell line‐derived neurotrophic factor (GDNF) for the treatment of Parkinson's disease constitutes one of the more promising clinical applications of neurotrophic factors. Crucial for clinical application will be the ability to deliver GDNF within the target structures, i.e. striatum and/or substantia nigra. We are developing both in vivo and ex vivo gene therapy approaches to reach this goal. We have shown in rodents that both lentiviral vectors coding for GDNF and polymer encapsulated cells genetically engineered to release GDNF are able to protect nigral dopaminergic neurons against various insults including axotomy and neurotoxins such as 6‐hydroxydopamine. Even more important for clinical application is the ability to scale‐up the technology to nonhuman primate application. Neurorestorative and/or neuroprotective properties of GDNF expression were demonstrated with both methods in various nonhuman primate models.