C12N2310/11

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.

Reprogramming of non-neuronal cells into neurons and methods and compositions to treat neurodegenerative diseases and disorders

Provided herein is a method of reprogramming a non-neuronal cell to a neuron. Aspects of the present disclosure relate to using cell reprogramming agent suppresses the expression or activity of PTB to convert a non-neuronal cell into a neuron. Also provided herein is a method of treating neurodegenerative disease by reprogramming non-neuronal cells in vivo to functional neurons.

ANTISENSE OLIGONUCLEOTIDES AND THIER USE FOR THE TREATMENT OF CANCER

The present invention concerns the treatment of prostate cancer and particularly castration resistant prostate cancer (CRPC). The Heat Shock Protein Hsp27, a chaperone protein, has been long demonstrated as a driver of Castration Resistance Prostate Cancer (CRPC). In the light of identification of the molecular mechanisms, the inventor determined that the Probable ATP-dependent RNA helicase DDX5 is an interactor of Hsp27 and DDX5's expression is modulated by Hsp27. They confirmed that DDX5 overexpression is correlated to the aggressiveness of the tumor, to the CRPC emergency and to the biochemical recurrence risk. They also developed DDX5-targeting antisense oligonucleotides for research purpose and clinical application. Thus, the invention relates to an inhibitor of DDX5 wherein said inhibitor reduces the expression and/or activity of DDX5 in a subject in need thereof and targets the gene or the mRNA of DDX5.

Methods and compositions for editing RNAs

The present application provides methods for editing RNA by introducing a deaminase-recruiting RNA in a host cell for deamination of an adenosine in a target RNA. The present application further provides deaminase-recruiting RNAs used in the RNA editing methods and compositions comprising the same.

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.

EXTRACELLULAR VESICLES WITH ANTISENSE OLIGONUCLEOTIDES TARGETING KRAS

The present disclosure relates to modified extracellular vesicles, e.g., exosomes, comprising an antisense oligonucleotide (ASO), which is capable of reducing and/or inhibiting expression of KRAS mRNA and/or KRAS protein. ASOs that can be used with the modified extracellular vesicles are also disclosed. Also provided herein are methods for using the exosomes and ASOs to treat and/or prevent diseases, such as cancer.

EXOSOMES-BASED THERAPY FOR LIVER FIBROSIS AND OTHER DISEASES WITH FIBROSIS

Provided herein are compositions of lipid-based nanoparticles, such as exosomes, that contain a therapeutic STAT3-targeting inhibitory RNA. Also provided are methods of using such compositions to treat a patient having fibrosis or a disease associated with fibrosis.

COMPOSITIONS FOR DELIVERY OF ANTISENSE COMPOUNDS

Provided herein are compounds comprising cyclic cell penetrating peptides and antisense compounds. Also provided herein are methods of modulating splicing, inhibiting or regulating translation, mediating degradation, blocking expansions of nucleotide repeats, and treating disease using the aforementioned compounds.

Optimized anti-FLT1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders

This disclosure relates to novel targets for angiogenic disorders. Novel oligonucleotides are also provided. Methods of using the novel oligonucleotides for the treatment of angiogenic disorders (e.g., preeclampsia) are also provided.

Inhibitor for use for preventing and/or treating an infection with hepatitis B virus

An inhibitor for use for preventing and/or treating an infection with hepatitis B virus (HBV) and an in vitro screening method for the identification of a candidate compound suitable for preventing and/or treating an infection with hepatitis B virus is provided.