Patent classifications
C12N2310/3231
COMPOUNDS AND METHODS FOR MODULATING FACTOR XII
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of FXII RNA in a cell or subject, and in certain instances reducing the amount of FXII protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to prevent, treat, or ameliorate at least one symptom of a thromboembolic condition. Such thromboembolic conditions include deep vein thrombosis, venous or arterial thrombosis, pulmonary embolism, myocardial infarction, and stroke. Such symptoms include pain, shortness of breath, heart burn, cold sweat, fatigue, lightheadedness, dizziness, swelling, cramping, and death. Such compounds, methods, and pharmaceutical compositions are useful to prevent, treat, or ameliorate at least one symptom of hereditary angioedema.
Antisense oligonucleotides targeting ATXN3
The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.
ANTISENSE OLIGONUCLEOTIDE SEQUENCES FOR SILENCING THE HUMAN L1-MET TRANSCRIPT IN TUMORS
The present invention concerns the use of antisense oligonucleotides to induce the death of several types of human cancer cells by silencing human L1-MET, which is a non coding transcript specifically transcribed in tumour cells.
LIBRARY OF BARCODED EXTRACELLULAR VESICLES
A factor that is caused by a nucleic acid and influences the kinetics of an extracellular vesicle is screened. A library of barcoded extracellular vesicles is provided.
Selective Reduction of Allelic Variants
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
Modulation of Angiopoietin-Like 3 Expression
Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof.
Modulation of Angiopoietin-Like 3 Expression
Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof.
RNA Interactome of Polycomb Repressive Complex 1 (PRC1)
This invention relates to polycomb-associated RNAs, libraries and fragments of those RNAs, inhibitory nucleic acids and methods and compositions for targeting RNAs, and methods of use thereof.
Oligonucleotide Progranulin Agonists
The present invention relates to oligonucleotides that upregulate or restore the expression of progranulin in cells by targeting the promoter region of the progranulin gene. The invention further relates to pharmaceutical compositions and methods for the treatment of diseases associated with progranulin, specifically progranulin haploinsufficiency and neurological disorders.
METHODS FOR DETECTING AN ANALYTE USING STRUCTURE SWITCHING BINDING AGENTS
The present invention relates to diagnostic test and technology. In particular, it relates to a method for determining an analyte suspected to be present in a sample comprising contacting said sample with at least one sensor element comprising at least one binding agent which is capable of specifically binding to the analyte and which comprises at least one magnetic label; and in functional proximity thereto a magnetic tunnel junction generating a signal which is altered upon binding of the analyte to the binding agent for a time and under conditions which allow for specific binding of the analyte suspected to be present in the sample to the at least one binding agent, measuring an altered signal generated by the magnetic tunnel junction upon analyte binding to the at least one binding agent comprising the at least one magnetic label, and determining the analyte based on the altered signal which is generated by the magnetic tunnel junction. The present invention further relates to a device for determining an analyte suspected to be present in a sample and for using such a device. Moreover, the present invention furthermore relates to an aptamer which is capable of specifically binding to an analyte and which comprises at least one magnetic label and a method for identifying such an aptamer. Finally, the invention relates to a kit for determining an analyte suspected to be present in a sample.