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INTRATHECAL ADMINISTRATION OF ADENO-ASSOCIATED-VIRAL VECTORS FOR GENE THERAPY.
INTRATHECAL ADMINISTRATION OF ADENO-ASSOCIATED-VIRAL VECTORS FOR GENE THERAPY.
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机译:鞘内注射病毒相关矢量的鞘内管理。
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摘要
A composition comprising at least one AAV vector formulated for intrathecal delivery to the central nervous system is described. The composition comprises at least one expression cassette which contains sequences encoding an immunoglobulin construct linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn).
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