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Gene therapy for glycogen storage diseases
专利权人:
Fondazione Telethon
发明人:
Parenti, Giancarlo,Ballabio, Andrea
申请号:
AU2013332061
公开号:
AU2013332061A1
申请日:
2013.10.17
申请国别(地区):
AU
年份:
2015
代理人:
摘要:
The present invention relates to a transcription factor EB (TFEB) protein, ortholog, recombinant or synthetic or biotechnological 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/

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