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INTRATHECAL ADMINISTRATION OF ADENO-ASSOCIATED-VIRAL VECTORS FOR GENE THERAPY
专利权人:
The Trustees of the University of Pennsylvania
发明人:
James M. Wilson,Christian Hinderer,William Thomas Rothwell
申请号:
US15771481
公开号:
US20180339065A1
申请日:
2016.10.27
申请国别(地区):
US
年份:
2018
代理人:
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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