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Recombinant Adeno-Associated virus (AAV) vector particles displaying high-affinity ligands for cell-type specific gene delivery
专利权人:
发明人:
申请号:
EP12005565.2
公开号:
EP2692731A1
申请日:
2012.07.31
申请国别(地区):
EP
年份:
2014
代理人:
摘要:
The present invention relates to recombinant adeno-associated virus (AAV) vector particles displaying high-affinity ligands, which can be used for cell-type specific gene delivery during therapeutic applications and applications in basic research, since they provide high cell-type selectivity or rather high targeting specificity, and to methods for their construction and generation.In detail, the present invention refers to a recombinant AAV vector particle, wherein the differences to the AAV wild-type particle are based on (a) at least one packaged transgene to be delivered to a specific target cell or target tissue, (b) a mutated AAV capsid protein VP2, wherein the mutation is based on a cell-type specific DARPin that is fused to the N-terminus of the capsid protein VP2, (c) a mutated VP2-start codon, and (d) a capsid, wherein the essential binding site for the natural receptor of the capsid proteins VP1, VP2 and VP3 is mutated.
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