您的位置: 首页 > 农业专利 > 详情页

GENE THERAPY FOR GLYCOGEN STORAGE DISEASES
专利权人:
FONDAZIONE TELETHON
发明人:
Giancarlo Parenti,Andrea Ballabio
申请号:
US14436161
公开号:
US20150273016A1
申请日:
2013.10.17
申请国别(地区):
US
年份:
2015
代理人:
摘要:
The present invention relates to a transcription factor EB (TFEB) protein, ortholog, recombinant or synthetic or bio technological functional derivative thereof, allelic variant thereof and fragments thereof; a chimeric molecule comprising the TFEB protein, ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a polynucleotide coding for said protein or ortholog, recombinant or synthetic or biotechnological functional derivative thereof, allelic variant thereof and fragments thereof; a vector comprising said polynucleotide; a host cell genetically engineered expressing said polypeptide or a pharmaceutical composition for use in the treatment or/and prevention of a glycogen storage disease. Preferably of Pompe or Danon disease.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

意 见 箱

匿名:登录

个人用户登录

找回密码

第三方账号登录

忘记密码

个人用户注册

必须为有效邮箱
6~16位数字与字母组合
6~16位数字与字母组合
请输入正确的手机号码

信息补充