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PROCÉDÉS D'ADMINISTRATION EFFICACE DE MOLÉCULES THÉRAPEUTIQUES IN VITRO ET IN VIVO
专利权人:
Massachusetts Eye & Ear Infirmary
发明人:
申请号:
EP15855354.5
公开号:
EP3212770A4
申请日:
2015.10.29
申请国别(地区):
EP
年份:
2018
代理人:
摘要:
Compositions are described for direct protein delivery into multiple cell types in the mammalian inner ear. The compositions are used to deliver protein(s) (such as gene editing factors) editing of genetic mutations associated with deafness or associated disorders thereof. The delivery of genome editing proteins for gene editing and correction of genetic mutations protect or restore hearing from genetic deafness. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.
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