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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:鞘内注射腺相关病毒载体进行基因治疗
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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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