Back to results
US 9,382,543 B2Granted
Treatment of pyrroline-5-carboxylate reductase 1 (PYCR1) related diseases by inhibition of natural antisense transcript to PYCR1
AssigneeCuRNA, Inc.
InventorsJoseph Collard, Olga Khorkova Sherman
Life of this patent
Estimating the term…
Abstract
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Pyrroline-5-carboxylate reductase 1 (PYCR1), in particular, by targeting natural antisense polynucleotides of Pyrroline-5-carboxylate reductase 1 (PYCR1). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of PYCR1.
Claims15
A synthetic, modified oligonucleotide of 14 to 30 nucleotides in length comprising at least one modification wherein the at least one modification is selected from:
- at least one modified sugar moiety;
- at least one modified internucleotide linkage;
- at least one modified nucleotide, and combinations thereof;
- wherein said oligonucleotide is an antisense compound which specifically hybridizes and shares 100% complementarity over the full length to a natural antisense polynucleotide of a Pyrroline-5-carboxylate reductase 1 (PYCR1) gene and upregulates the function and/or expression of a Pyrroline-5-carboxylate reductase 1 (PYCR1) gene in vivo or in vitro as compared to a normal control.
1 more element
Dependencies are inferred from the claim text.