Title:
止血疾患を治療するための変異因子ＶＩＩＩのゲノム組込及び発現のための改良された発現カセット
Document Type and Number:
Japanese Patent JP7237903
Kind Code:
B2
Abstract:
Factor VIII variants and methods of use thereof are disclosed. In particular embodiments, Factor VIII variants are expressed more efficiently by cells over wild-type Factor VIII proteins, are secreted at increased levels by cells over wild-type Factor VIII proteins, exhibit enhanced activity over wild-type Factor VIII proteins and are packaged more efficiently into viral vectors.
Inventors:
Sabatino, Dennis
Hi, Catherine A.
El Carbie, Lyron
Hi, Catherine A.
El Carbie, Lyron
Application Number:
JP2020153781A
Publication Date:
March 13, 2023
Filing Date:
September 14, 2020
Export Citation:
Assignee:
The Children's Hospital of Philadelphia
International Classes:
C12N15/12; A61K35/76; A61K35/761; A61K38/37; A61K48/00; A61P7/02; A61P7/04; A61P9/00; A61P17/02; C07K14/755; C12N5/10; C12N15/85; C12N15/864; C12P21/02
Foreign References:
| WO2011005968A1 | ||||
| WO2014064277A1 | ||||
| US20120308641 |
Other References:
Nucleic Acids Research,1991年,Vol.19, No.5,p4139-4145
Attorney, Agent or Firm:
Satoshi Murakoshi