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4 results about "Antisense RNA" patented technology
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Antisense RNA (asRNA), also referred to as antisense transcript, natural antisense transcript (NAT) or antisense oligonucleotide, is a single stranded RNA that is complementary to a protein coding messenger RNA (mRNA) with which it hybridizes, and thereby blocks its translation into protein. asRNAs (which occur naturally) have been found in both prokaryotes and eukaryotes, antisense transcripts can be classified into short (<200 nucleotides) and long (>200 nucleotides) non-coding RNAs (ncRNAs). The primary function of asRNA is regulating gene expression. asRNAs may also be produced synthetically and have found wide spread use as research tools for gene knockdown. They may also have therapeutic applications.
The present invention comprises a pharmaceutic composition comprising one or more hepatocyte nuclear factor four alpha antisense RNA 1 (HNF4A-AS1) targeting polynucleotides, wherein the one or more HNF4A-AS1 targeting polynucleotides is capable of repressing HNF4A-AS1 in a subject resulting in upregulation of HNF4A expression and / or increasing HNF4A P1:P2 isoform ratio in the subject. The present invention also provides a method of treatment of a HNF4A-associated disease in a subject comprising the step of administering a therapeutically effective amount of the pharmaceutical composition comprising one or more HNF4A- AS1 targeting polynucleotides of the present invention. The present invention also provides a method of downregulating HNF4A P2 isoform expression in a subject comprising the step of repressing HNF4A-AS1 in the subject.
The present invention provides a composition comprising one or more polynucleotides capable of upregulating expression of alpha- 1 anti-trypsin (Al AT). In an embodiment, the one or more polynucleotides of the present invention upregulates the expression of Al AT by downregulating antisense RNA that regulates the Al AT gene. In an embodiment, the one or more polynucleotides comprises an siRNA or an shRNA. In an embodiment, one or more polynucleotides of the present invention is delivered by a nanoparticle such as an exosome that encapsulates the one or more polynucleotides. The present invention also provides a method of treatment of Al AT deficiency related diseases or disorders using any embodiment of the one or more polynucleotides of the present invention.