Patent classifications
C12N2310/141
MicroRNA (miRNA) and Downstream Targets for Diagnostic and Therapeutic Purposes
In some embodiments, the invention is directed to a method for diagnosing fibrosis and/or fibrosis related diseases and to a method for screening a pharmaceutically active compound for the treatment of fibrosis and/or fibrosis related diseases. The present invention further relates to compositions for use in the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions modulate the activity of a miRNA for the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions inhibit the activity of miR-21 for the treatment, amelioration, and/or prevention of fibrosis.
FMRP AND CANCER TREATMENT
The present invention provides compositions and methods for down-modulating the expression and/or the immuno-suppressive activity of i) the FMRP protein, ii) an mRNA encoding the FMRP protein, and/or iii) the FMR1 gene for the treatment and/or prevention of primary cancer and/or cancer metastasis in a subject in need thereof.
RNA INTERFERENCE-INDUCING NUCLEIC ACID COMPRISING 8-OXOGUANINE, MODIFIED NUCLEIC ACID BINDING TO MICRORNA COMPRISING 8-OXOGUANINE, AND USES THEREOF
In the present invention, it has been confirmed that, when an RNA interference-inducing nucleic acid including at least one 8-oxoguanine (o.sup.8G) in 1st to 9th nucleotides from the 5′-end of at least one single strand of a double strand of a nucleic acid, and a modified nucleic acid that specifically binds to microRNA and in which at least one guanine (G) from among the 1st to 9th nucleotides from the 5′-end are modified with 8-oxoguanine (o.sup.8G), are produced and administered to cells or mice, various pathophysiological phenomena are induced.
In addition, the positions where G>T modifications occur have been identified in cDNA produced through the reverse transcription of microRNA in which guanine (G) is oxidatively modified with 8-oxoguanine (o.sup.8G) by oxidative stress in a seed region of microRNA, to confirm the positions where oxidative modification to 8-oxoguanine has occurred.
COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE COMPRISING INHIBITOR OF ATLASTIN 2, AND METHOD FOR DIAGNOSING ALZHEIMER'S DISEASE BY DETERMINING ATLASTIN 2
The present invention relates to a composition for preventing or treating Alzheimer’s disease, containing an inhibitor of ATL2, and a method of diagnosing the disease based on the measurement of the ATL2. In the present invention, it was found that PS1 mutants may result in mitochondrial dysfunction, such as increased binding between endoplasmic reticulum and mitochondria, increased mitochondrial ROS production, decreased mitochondrial membrane potential, decreased ATP production, decreased complex I activity, and decreased peroxidase activity, in brain glioma cells and that the PS1 mutants may abnormally increase the binding between endoplasmic reticulum and mitochondria by elevating the expression of the ATL2 in the brain. In addition, when the ATL2 was knocked down, it was observed that the binding between endoplasmic reticulum and mitochondria was lowered and that the expression of the ATL2 was elevated in the brains of Alzheimer’s disease animal models and patients. Accordingly, it is expected that it may possible to effectively prevent or treat Alzheimer’s disease by inhibiting the expression or activity of the ATL2 and that it may possible to diagnose the disease, predict the risk of developing the disease, and screen therapeutic agents for the disease, by measuring the level of the expression or activity of the ATL2.
High cAMP yielding yeast strain and use thereof
Provided is a yeast strain capable of excessively synthesizing cAMP and its construction method and fermentation technique thereof, and application in the field of medicine, animal husbandry, food or chemical industry. The yeast strain includes first and second gene modifications, wherein the first gene includes protein kinase A (PKA) catalytic subunit encoding genes TPK1, TPK2 and TPK3, by modifying the first gene, the activity or expression of PKA is completely inhibited, so that feedback inhibition to cyclic adenosine monophosphate (cAMP) is eliminated, but at the same time, the growth of the yeast is inhibited; and the second gene modification eliminates growth inhibition caused by the first gene modification, so that the yeast grows normally, and the cAMP yield by the yeast is increased, wherein the increase of the cAMP yield is relative to the cAMP yield by an unmodified yeast. The yeast strain further includes third and/or fourth gene modifications. The recombinant yeast strain of the present invention can stably, continuously and efficiently produce extracellular cAMP by up to 9721.6 μmol/L.
In vitro and in vivo intracellular delivery of siRNA via self-assembled nanopieces
The compositions and methods of the invention provide compositions and methods for preferential targeting of tissues to delivery therapeutic or diagnostic agents. For example, such compounds are useful in the treatment of joint disorders those affecting articulating joints, e.g., injury-induced osteoarthritis as well as autoimmune diseases affecting joint tissue such as rheumatoid arthritis.
TRNA/pre-miRNA compositions and methods for treating hepatocellular carcinoma
Provided are hybrid tRNA/pre-microRNA and tRNA molecules and their use in methods of preventing and treating hepatocellular carcinoma (HCC). In some embodiments, provided are polynucleotides that include a tRNA operably linked to one or more pre-microRNA (pre-miRNA), where the tRNA and/or pre-miRNA are operably linked to one or more inserted RNA molecules that inhibit the growth or proliferation of a hepatocellular carcinoma (HCC) cell.
SIGNAL-SENSOR POLYNUCLEOTIDES FOR THE ALTERATION OF CELLULAR PHENOTYPES
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of signal-sensor polynucleotides, primary transcripts and mmRNA molecules.
AN UBIQUITIN LIGASE INHIBITOR FOR USE FOR PREVENTING AND/OR TREATING A DISEASE LINKED WITH CEREBRAL HYPOPERFUSION
The present invention concerns an ubiquitin ligase inhibitor for use for preventing and/or treating a disease linked with cerebral hypoperfusion, and an in vitro screening method for the identification of a candidate compound suitable for preventing and/or treating a disease linked with cerebral hypoperfusion.
METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
Compositions and methods for modulating expression of target nucleic acids using a single strand oligoribonucleotide ss-siRNA compound are disclosed.