TREATMENT OF PYRROLINE-5-CARBOXYLATE REDUCTASE 1 (PYCR1) RELATED DISEASE BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO PYCR1
First Claim
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1. A method of identifying an antisense oligonucleotide which can modulate a function of and/or the expression of a Pyrroline-5-carboxylate reductase 1 (PYCR1) polynucleotide in patient cells or tissues in vivo or in vitro comprising:
- identifying at least one antisense oligonucleotide of 10 to 30 nucleotides in length which is at least 90% complementary to a region of a polynucleotide antisense to an RNA transcribed from the PYCR1 polynucleotide;
contacting said at least one antisense oligonucleotide with said polynucleotide antisense to said RNA transcribed from the PYCR1 polynucleotide to form a hybrid;
measuring the thermal melting point of the hybrid of the at least one antisense oligonucleotide with said polynucleotide antisense to said RNA under stringent hybridization conditions;
selecting at least one antisense oligonucleotide having binding affinity to said polynucleotide based upon the thermal melting point results;
determining the modulation activity of the selected at least one antisense oligonucleotide targeted to said polynucleotide antisense to the RNA transcribed from the PYCR1 polynucleotide andidentifying at least one antisense oligonucleotide which modulates a function of and/or expression of the PYCR1 polynucleotide.
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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.
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15 Claims
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1. A method of identifying an antisense oligonucleotide which can modulate a function of and/or the expression of a Pyrroline-5-carboxylate reductase 1 (PYCR1) polynucleotide in patient cells or tissues in vivo or in vitro comprising:
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identifying at least one antisense oligonucleotide of 10 to 30 nucleotides in length which is at least 90% complementary to a region of a polynucleotide antisense to an RNA transcribed from the PYCR1 polynucleotide; contacting said at least one antisense oligonucleotide with said polynucleotide antisense to said RNA transcribed from the PYCR1 polynucleotide to form a hybrid; measuring the thermal melting point of the hybrid of the at least one antisense oligonucleotide with said polynucleotide antisense to said RNA under stringent hybridization conditions; selecting at least one antisense oligonucleotide having binding affinity to said polynucleotide based upon the thermal melting point results; determining the modulation activity of the selected at least one antisense oligonucleotide targeted to said polynucleotide antisense to the RNA transcribed from the PYCR1 polynucleotide and identifying at least one antisense oligonucleotide which modulates a function of and/or expression of the PYCR1 polynucleotide. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15)
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