C/EBPα short activating RNA compositions and methods of use
The invention relates to saRNA targeting a C/EBPα transcript and therapeutic compositions comprising said saRNA. Methods of using the therapeutic compositions are also provided.
1. A synthetic short activating RNA (saRNA) which up-regulates the expression of a target gene, wherein the target gene is CCAAT/enhancer-binding protein alpha (CEBPA), wherein the saRNA comprises an anti-sense strand of 19-21 nucleotides, wherein the antisense strand comprises a sequence of GACCAGUGACAAUGACCGC (SEQ ID No: 385), and wherein the antisense strand comprises at least one unpaired nucleotide at the 3′ end forming 3′ overhangs.
2. The saRNA of claim 1 , wherein the antisense strand comprises 2 unpaired nucleotides at the 3′ end forming 3′ overhangs.
3. The saRNA of claim 1 , wherein the antisense strand comprises at least one chemical modification.
4. The saRNA of claim 1 , wherein the antisense strand has a sequence of SEQ ID No: 318 or 374.
5. The saRNA of claim 1 , wherein the saRNA is double stranded and further comprises a sense strand.
6. The saRNA of claim 5 , wherein the sense strand comprises at least one chemical modification.
7. The saRNA of claim 5 , wherein the sense strand has a sequence selected from SEQ ID No: 280 or 364.
8. The saRNA of claim 1 , wherein the saRNA is double stranded and further comprises a sense strand, wherein the antisense strand comprises SEQ ID No. 318 and the sense strand comprises SEQ ID No. 280.
9. The saRNA of claim 1 , wherein the saRNA is double stranded and further comprises a sense strand, wherein the antisense strand comprises SEQ ID No. 374 and the sense strand comprises SEQ ID No. 364.
10. The saRNA of claim 1 , wherein the unpaired nucleotide is a uracil.
11. The saRNA of claim 2 , wherein the unpaired nucleotides are uracils.