发明名称 |
EFFICIENT DELIVERY OF THERAPEUTIC MOLECULES IN VITRO AND IN VIVO |
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. |
申请公布号 |
WO2016069912(A1) |
申请公布日期 |
2016.05.06 |
申请号 |
WO2015US58096 |
申请日期 |
2015.10.29 |
申请人 |
MASSACHUSETTS EYE AND EAR INFIRMARY |
发明人 |
CHEN, ZHENG-YI;LIU, DAVID |
分类号 |
A61K38/46;C07H21/04;C07K14/00;C12N5/00;C12N9/16;C12P21/04 |
主分类号 |
A61K38/46 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|