Clinical applications involving CNS gene transfer.
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ABSTRACT:
SUBMITTER: Kantor B
PROVIDER: S-EPMC4518844 | biostudies-literature | 2014
REPOSITORIES: biostudies-literature
Publications
Clinical applications involving CNS gene transfer.
Kantor Boris B McCown Thomas T Leone Paola P Gray Steven J SJ
Advances in genetics 20140101
Diseases of the central nervous system (CNS) have traditionally been the most difficult to treat by traditional pharmacological methods, due mostly to the blood-brain barrier and the difficulties associated with repeated drug administration targeting the CNS. Viral vector gene transfer represents a way to permanently provide a therapeutic protein within the nervous system after a single administration, whether this be a gene replacement strategy for an inherited disorder or a disease-modifying p ...[more]
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