基本信息:
- 专利标题： METHODS AND MATERIALS FOR TREATING HUNTINGTON'S DISEASE
- 申请号：PCT/US2020/038050 申请日：2020-06-17
- 公开(公告)号：WO2020263639A1 公开(公告)日：2020-12-30
- 发明人： CHEN, Gong , WU, Zheng , GUO, Ziyuan , CHEN, Yuchen , PEI, Zifei
- 申请人： THE PENN STATE RESEARCH FOUNDATION
- 申请人地址： 304 Old Main Street
- 专利权人： THE PENN STATE RESEARCH FOUNDATION
- 当前专利权人： THE PENN STATE RESEARCH FOUNDATION
- 当前专利权人地址： 304 Old Main Street
- 代理机构： WILLIS, Margaret, S. et al.
- 优先权： US62/868,499 2019-06-28
- 主分类号： A61P25/28
- IPC分类号： A61P25/28 ; C07K14/475 ; C12N5/00 ; C12N9/22 ; A01K2227/105 ; A01K2267/0318 ; A61K38/02 ; A61K38/1709 ; A61K48/00 ; A61K48/005 ; A61K9/0085 ; A61P43/00 ; C12N15/86 ; C12N15/907 ; C12N2501/195 ; C12N2506/08 ; C12N2510/00 ; C12N2750/14143 ; C12N5/0619 ; C12N5/0622
摘要：
This document provides methods and materials for treating a mammal having Huntington's disease. For example, methods and materials for forming GABAergic neurons that are functionally integrated into the brain of a living mammal (e.g., a human) and/or for modifying one or both huntingtin (Htt) genes (or HTT RNAs or HTT polypeptides) present in a mammal with Huntington's disease are provided.
IPC结构图谱:
| A | 人类生活必需 |
| --A61 | 医学或兽医学；卫生学 |
| ----A61P | 化合物或药物制剂的治疗活性 |
| ------A61P25/00 | 治疗神经系统疾病的药物 |
| --------A61P25/28 | .用于治疗中枢神经系统神经变性疾病的药物，例如精神功能改善剂、识别增强剂、用于治疗早老性痴呆或其他类型的痴呆的药物 |