Patent classifications
C12N2310/316
Selective Antisense Compounds and Uses Thereof
The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
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 ?-thalassemia or hemochromatosis.
Treatment of uncoupling protein 2 (UCP2) related diseases by inhibition of natural antisense transcript to UCP2
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Uncoupling Protein 2 (UCP2), in particular, by targeting natural antisense polynucleotides of Uncoupling Protein 2 (UCP2). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of UCP2.
TREATMENT OF GLIAL CELL DERIVED NEUROTROPHIC FACTOR (GDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO GDNF
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Glial cell derived neurotrophic factor (GDNF), in particular, by targeting natural antisense polynucleotides of Glial cell derived neurotrophic factor (GDNF). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of GDNF.
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 ?-thalassemia or hemochromatosis.
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.
MODULATION OF NOX4 EXPRESSION
Provided herein are methods, antisense agents, specific inhibitors, and compositions useful for reducing expression or activity of NADPH oxidase 4 (hereinafter referred to as NOX4) in a subject. Also, provided herein are methods, antisense agents, specific inhibitors, and compositions that can be useful in treating NOX4-related diseases or conditions in a subject. Such methods, antisense agents, specific inhibitors, and compositions can be useful, for example, to treat a pulmonary disease in a subject.
METHODS OF PLACING LOCKED NUCLEIC ACIDS IN SMALL INTERFERING RNA STRANDS
Provided herein include methods, systems, and compositions for placing locked nucleic acids in small interfering RNA (siRNA) strands, for example conditionally activatable siRNA sensor strands, as well as the siRNA complexes generated using the method herein described and the component strands. The siRNA complex can be conditionally activated upon a complementary binding to an input nucleic acid strand (e.g. a mRNA of a biomarker gene specific to a target cell) through a sequence in a sensor nucleic acid strand of the nucleic acid complex. The activated nucleic acid complex can release a RNAi duplex which can specifically inhibit a target RNA.
Method of promoting wound healing
Disclosed is a method of promoting wound healing or wound closure. The method comprises administration of a miR-198 inhibitor and/or a follistatin-like-1 (FSTL1) polypeptide. Also disclosed are method of treating chronic cutaneous wounds, method of identifying a non-healing wound, use and a pharmaceutical composition comprising a miR-198 inhibitor and/or a follistatin-like-1 (FSTL1) polypeptide.
TREATMENT OF REPROGRAMMING FACTOR RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO A REPROGRAMMING FACTOR
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Reprogramming factor, in particular, by targeting natural antisense polynucleotides of a Reprogramming factor. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Reprogramming factors.