Potential of gene therapy for pediatric neurotransmitter diseases: lessons from Parkinson's disease.

Abstract:

: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.

journal_name

Ann Neurol

journal_title

Annals of neurology

authors

Kang UJ,Nakamura K

doi

10.1002/ana.10654

keywords:

subject

Has Abstract

pub_date

2003-01-01 00:00:00

pages

S103-9

eissn

0364-5134

issn

1531-8249

journal_volume

54 Suppl 6

pub_type

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