COMPOSITIONS AND METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS

Invention Application

US20180100144A1 COMPOSITIONS AND METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS 审中-公开

Patent Title: COMPOSITIONS AND METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS
Application No.: US15569203

Application Date: 2016-04-27
Publication No.: US20180100144A1

Publication Date: 2018-04-12
Inventor: Ana Maria BUJ BELLO , Mirella LO SCRUDATO
Applicant: GENETHON , INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , UNIVERSITE D'EVRY VAL D'ESSONNE
Priority: EPEP15305646.0 20150427
International Application: PCT/EP2016/059346 WO 20160427
Main IPC: C12N15/10
IPC: C12N15/10 ; C12N15/11

Abstract:

The present invention relates to compositions and methods for the treatment of nucleotide repeat expansion disorders such as myotonic dystrophy.

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IPC分类:

C	化学；冶金
C12	生物化学；啤酒；烈性酒；果汁酒；醋；微生物学；酶学；突变或遗传工程
C12N	微生物或酶；其组合物；繁殖、保藏或维持微生物；变异或遗传工程；培养基（微生物学的试验介质入C12Q1/00）。
C12N15/00	突变或遗传工程；遗传工程涉及的DNA或RNA，载体（如质粒）或其分离、制备或纯化；所使用的宿主（突变体或遗传工程制备的微生物本身入C12N1/00、C12N5/00、C12N7/00；新的植物入A01H；用组织培养技术再生植物入A01H4/00；新的动物入A01K67/00；含有插入活体细胞的遗传物质以治疗遗传疾病的药剂的应用，基因疗法入A61K48/00，一般肽入C07K）
C12N15/09	.DNA重组技术
C12N15/10	..分离、制备或纯化DNA或RNA的方法（DNA或RNA的化学制备入C07H21/00；自微生物中或用酶制备非结构多核苷酸入C12P19/34）