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Potential of gene therapy for pediatric neurotransmitter diseases: Lessons from Parkinson's disease
Author(s) -
Kang Un Jung,
Nakamura Ken
Publication year - 2003
Publication title -
annals of neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.764
H-Index - 296
eISSN - 1531-8249
pISSN - 0364-5134
DOI - 10.1002/ana.10654
Subject(s) - genetic enhancement , neurotransmitter , parkinson's disease , disease , neurotransmitter systems , neuroscience , neurotrophic factors , bioinformatics , medicine , neurotransmitter agents , neurotransmitter receptor , degenerative disease , gene , biology , central nervous system , genetics , dopamine , pathology , receptor
Gene therapy methods have continued to develop rapidly, and many initial limitations that hampered clinical application have been overcome. Thus serious consideration of clinical application of gene therapy is warranted for selected disorders in which the pathogenesis is well defined. Parkinson's disease has been the most extensively studied target of gene therapy for central nervous system disorders and shares many features with pediatric neurotransmitter diseases. Neurotransmitter replacement therapy using catecholamine‐synthesizing genes and delivery of neurotrophic factors such as glial cell line‐derived neurotrophic factors has been successful in animal models of Parkinson's disease. Application of gene therapy for pediatric neurotransmitter diseases will require delineating the optimal set of genes to correct the consequences of the deficiencies. The optimal anatomical targets and proper timing of the gene replacement must be understood. Safety of gene therapy vehicles and the ability to regulate gene expression will be essential for eventual clinical application. Ann Neurol 2003;54 (suppl 6):S103–S109