Thierry VANDENDRIESSCHE,Marinee CHUAH,J. Keith JOUNG,Yanfang FU,Deepak REYON
申请号：
US15311579
公开号：
US20170088819A1
申请日:
2015.05.18
申请国别(地区):
US
年份:
2017
代理人:
摘要：
The invention relates to polynucleotides suitable for reducing or eliminating the expression of expanded repeat RNA (CUGexp) of the dystrophy myotonic-protein kinase (DMPK) gene in a cell of a DM-1 patient. The polynucleotides are a combination of a polynucleotide for a site specific nuclease targeting the dystrophy myotonic-protein kinase (DMPK) gene locus, and a donor polynucleotide having 5′ and 3′ regions which are homologous with the sequence of DMPK gene which flank the target site of the nuclease. The invention further relate to in vivo and in vitro methods to reduce or eliminate CTG repeats in the DMPK gene. The invention further relates to the medical use of polynucleotides and cells for treating DM-1 patient.
