Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
First Claim
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1. A method of upregulating a function of and/or the expression of a RCAN1 polynucleotide in patient cells or tissues in vitro comprising:
- contacting said cells or tissues with at least one single stranded antisense oligonucleotide of about 10 to 30 nucleotides in length that targets a 10 to 30 consecutive nucleotide region of a natural antisense oligonucleotide of the RCAN1 polynucleotide selected from SEQ ID NOS;
5 or 6;
thereby upregulating the function of and/or the expression of the RCAN1 Gene polynucleotide in patient cells or tissues in vitro.
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Abstract
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Down Syndrome Gene, in particular, by targeting natural antisense polynucleotides of a Down Syndrome Gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Down Syndrome Genes.
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11 Claims
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1. A method of upregulating a function of and/or the expression of a RCAN1 polynucleotide in patient cells or tissues in vitro comprising:
contacting said cells or tissues with at least one single stranded antisense oligonucleotide of about 10 to 30 nucleotides in length that targets a 10 to 30 consecutive nucleotide region of a natural antisense oligonucleotide of the RCAN1 polynucleotide selected from SEQ ID NOS;
5 or 6;
thereby upregulating the function of and/or the expression of the RCAN1 Gene polynucleotide in patient cells or tissues in vitro.- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10)
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11. A method of upregulating a function of and/or the expression of a RCAN1 polynucleotide in mammalian cells or tissues in vitro comprising:
contacting said cells or tissues with at least one short interfering RNA (siRNA) oligonucleotide 19 to 30 nucleotides in length, said at least one siRNA oligonucleotide being specific for a natural antisense polynucleotide of the RCAN1 polynucleotide, Wherein said natural antisense polynucleotide is selected from SEQ ID NOS;
5 or 6 or a natural antisense polynucleotide antisense to non-coding regions of an RNA transcribed from the RCAN1 gene and, upregulating the function of and/or the expression of the RCAN1 polynucleotide in mammalian cells or tissues in vitro.
Specification