C12N2310/336

RNA INTERFERENCE-INDUCING NUCLEIC ACID INHIBITING NONCANONICAL TARGETS OF MICRO RNA, AND USE FOR SAME

The present invention relates to RNA interference-inducing nucleic acid that inhibits noncanonical target genes of micro RNA, in which part of the sequence of a specific micro RNA has been modified, and by using the RNA interference-inducing nucleic acid of the present invention, the biological function micro RNA exhibits by inhibiting noncanonical target genes is effectively increased or there is the benefit of selectively exhibiting only one of the biological functions of conventional micro RNA, i.e., the function of inhibiting noncanonical target genes, and the interference-inducing nucleic acid of the present invention enables cell cycling, differentiation, dedifferentiation, formation, movement, splitting, proliferation or death adjustment, and it is expected that the invention can be used in various fields such as drugs and cosmetics.

Secretory immunoglobulin a (sIgA)-binding nucleic acid molecule, sIgA analysis sensor, and sIgA analysis method

The present invention provides a novel molecule that can be used for detection of sIgA. The sIgA-binding nucleic acid molecule of the present invention is characterized in that it binds to secretory immunoglobulin A (sIgA) with a dissociation constant of 37.7 nM or less, and preferably includes a polynucleotide consisting of any of base sequences of SEQ ID NOs: 1 to 12 or a partial sequence thereof, for example. According to the sIgA-binding nucleic acid molecule of the present invention, it is possible to detect sIgA in saliva.

Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use

RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.

Pharmaceutical composition containing a stabilised mRNA optimised for translation in its coding regions

The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilised by sequence modifications and optimised for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilisation and translational efficiency of modified mRNA of the invention.

Exponential base-3 and greater nucleic acid amplification with reduced amplification time
11352622 · 2022-06-07 · ·

Described herein are methods and compositions that provide highly efficient nucleic acid amplification. In some embodiments, this allows a 3-fold or greater increase of amplification product for each amplification cycle and therefore increased sensitivity and speed over conventional PCR. Modified bases can be employed in primers to provide this base-3 or greater amplification with satisfactory PCR cycle times, which are improved, as compared to those observed in the absence of modified bases.

RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.

ALPHA-1 ANTITRYPSIN (AAT) RNAI AGENTS, COMPOSITIONS INCLUDING AAT RNAI AGENTS, AND METHODS OF USE

RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.

Modified compounds and uses thereof

The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.

HIF 1-alpha antisense oligonucleotides

Provided are peptide nucleic acid derivatives targeting a part of the human HIF-1α pre-mRNA. The peptide nucleic acid derivatives potently induce exon skipping to yield splice variants of HIF-1α mRNA in cells, and are useful to treat indications or conditions involving the overexpression of HIF-1α.

Immunostimulatory sequence oligonucleotides and methods of using the same

The invention provides immunomodulatory polynucleotides and methods for immunomodulation of individuals using the immunomodulatory polynucleotides.