Patent classifications
C12N2320/31
Targeted Inhibition Using Engineered Oligonucleotides
Disclosed herein are engineered oligonucleotides for selective inhibition of polypeptide expression and activity. Also disclosed herein are methods of selectively inhibiting polypeptide expression and activity contacting an engineered oligonucleotide with a polynucleotide encoding the polypeptide.
COMPOSITIONS AND METHODS OF TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
UTILIZING RNA INTERFERENCE AGAINST SARS-COV-2
An RNAi molecule, the RNAi molecule, vector and composition including the RNAi molecule and method of using same, wherein the RNAi molecule includes a dsRNA sequence of from about 15 to about 60 base pairs, capable of decreasing expression of a SARS-CoV-2 gene.
USE OF RAD18 INHIBITORS IN THE TREATMENT OF TUMORS
The present invention relates to the use of an inhibitor of Rad18 expression or activity, in treating a tumor or in sensitizing a patient affected with a tumor, to a treatment with an antineoplastic agent that is a DNA damaging chemotherapeutic agent so to both reduce the self renewal of cancer stem cells and increase the DNA damage response thus boosting apoptotic cell death.
TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as β-thalassemia or hemochromatosis.
ANTI-ARRHYTHMICITY AGENTS
Agents, compositions, and methods for regulating cardiac rhythmicity are disclosed.
ANTICANCER THERAPEUTIC INTERVENTION
The present invention is directed to a method of treating cancer using interfering RNA duplexes to mediate gene silencing. The present invention is also directed to interfering RNA duplexes and vectors encoding such interfering RNA duplexes.
HUMAN ANTIBODIES TO THE GLUCAGON RECEPTOR
The present invention provides antibodies that bind to the human glucagon receptor, designated GCGR and methods of using same. According to certain embodiments of the invention, the antibodies are fully human antibodies that bind to human GCGR. The antibodies of the invention are useful for lowering blood glucose levels and blood ketone levels and are also useful for the treatment of diseases and disorders associated with one or more GCGR biological activities, including the treatment of diabetes, diabetic ketoacidosis and long-term complications associated with diabetes, or other metabolic disorders characterized in part by elevated blood glucose levels.
COMPOSITION OF DRUG TARGETS AND METHOD OF USING THEREOF
A composition of drug targets and the use the method of using thereof. The composition comprises a vector and a drug using FKBP10 and PCOLCE genes and/or the encoded proteins thereof as drug targets.
Compositions and methods for treating pancreatic cancer
The present disclosure provides compositions and methods for treating or reducing the risk of pancreatic cancer by administering compounds capable of inhibiting the expression or activity of RUNX3.