| 发明名称 |
COMPOSITIONS AND METHODS FOR MODULATING SMN GENE FAMILY EXPRESSION |
| 摘要 |
Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2. |
| 申请公布号 |
US2015252364(A1) |
申请公布日期 |
2015.09.10 |
| 申请号 |
US201314401194 |
申请日期 |
2013.05.16 |
| 申请人 |
RaNA Therapeutics, Inc. ;The General Hospital Corporation d/b/a Massachusetts General Hospital |
发明人 |
Krieg Arthur M.;Subramanian Romesh;McSwiggen James;Lee Jeannie T. |
| 分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A single stranded oligonucleotide having a sequence 5′-X-Y-Z, wherein X is any nucleotide, Y is a nucleotide sequence of 6 nucleotides in length that is not a seed sequence of a human microRNA, and Z is a nucleotide sequence of 1-23 nucleotides in length, wherein the single stranded oligonucleotide is complementary with at least 8 consecutive nucleotides of a PRC2-associated region of an SMN gene. |
| 地址 |
Cambridge MA US |
