发明名称 |
Methods of inducing insulin production |
摘要 |
The present invention provides a method of inducing insulin production in a cell by up-regulating a target gene involved in insulin production in said cell using an saRNA (short activating ribonucleic acid) which specifically down-regulates a target antisense RNA transcript present in said cell, wherein (i) said target RNA transcript is complementary to a sequence located on the coding strand of the target gene between 1000 nucleotides upstream and 1000 nucleotides downstream of the transcription start site of the target gene; and (ii) said sRNA is a single or double stranded RNA molecule up to 30 nucleotides in length comprising a sequence of at least 13 nucleotides which has at least 95% complementarity to a region of the target transcript. Also provided are certain saRNA molecules and uses thereof, particular medical uses, and induced cells and uses thereof. |
申请公布号 |
US8916534(B2) |
申请公布日期 |
2014.12.23 |
申请号 |
US201113877968 |
申请日期 |
2011.10.10 |
申请人 |
Mina Therapeutics Limited |
发明人 |
Saetrom Pal |
分类号 |
C07H21/02;C07H21/04;A61K31/70;C12N15/113;C12N15/11;C12Q1/68 |
主分类号 |
C07H21/02 |
代理机构 |
DT Ward P.C. |
代理人 |
Ward Donna T.;Zhu Heng;DT Ward P.C. |
主权项 |
1. A short activating ribonucleic acid (saRNA) which upregulates PDX1 expression in a cell, said saRNA having a first strand consisting of (i) a sequence selected from SEQ ID NO: 15, 16, 17, and 18; and (ii) a 3′ tail formed of 1-5 uracils. |
地址 |
London GB |