Treatment of ABCA1 gene related diseases by inhibition of a natural antisense transcript to ABCA1
AssigneeCuRNA, Inc.
InventorsJoseph Collard, Olga Khorkova Sherman
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Abstract
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Lipid transport and metabolism gene, in particular, by targeting natural antisense polynucleotides of a Lipid transport and metabolism 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 a Lipid transport and metabolism genes.
Claims12
A method of upregulating a function of and/or the expression of an ABCA1 polynucleotide having SEQ ID NO:
- 1 in patient cells or tissues in vivo or in sum comprising:
- contacting said cells or tissues with at least one modified single stranded antisense compound of 12 to 30 nucleotides in length that targets and specifically hybridizes to a complementary region of a natural antisense polynucleotide of the ABCA1 polynucleotide having SEQ ID NO:
- 8 or 9;
- thereby upregulating function of and/or the expression of the ABCA1 polynucleotide in patient cells or tissues in vivo or in vitro.
A method of preventing or treating a disease associated with lipid transport or metabolism and insufficient expression of an ABCA1 gene product, comprising:
- administering to a patient a therapeutically effective dose of at least one modified single stranded antisense compound of 12 to 30 nucleotides in length that binds and specifically hybridizes to a natural antisense sequence of said ABCA1 gene polynucleotide having SEQ ID NO:
- 8 and upregulates expression of said ABCA1 gene polynucleotide;
- thereby treating the disease associated with the ABCA1 gene polynucleotide and/or at least one encoded product thereof wherein said diseases are selected from the group comprising a cardiovascular disorder or disease or condition associated with the ABCA1 gene polynucleotide wherein the disease associated with the ABCA1 gene polynucleotide is selected from:
- diabetes, obesity, dyslipidemia, hyperglycemia, hyperinsulinemia, hypercholesterolemia, atherosclerosis, familial HDL deficiency (FHD), Sea-blue histiocytosis Tangier's Disease, Fish-eye disease, LCAT deficiency or low-HDL cholesterolemia.
Dependencies are inferred from the claim text.