Patent classifications
C12Y101/03015
NUCLEIC ACIDS CONTAINING ABASIC NUCLEOTIDES
The present invention relates to nucleic acid molecules for use in the treatment or prevention of disease.
Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, HAO1) and methods of use thereof
The invention relates to polynucleotide agents targeting an hydroxyacid oxidase (HAO1) gene, and methods of using such polynucleotide agents to inhibit expression of HAO1 and to treat subjects having an HAO1-associated disease, e.g., hyperoxaluria.
COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
Methods and microorganisms for the production of glycolic acid and/or glyoxylic acid
The present invention relates to methods and recombinant microorganisms useful for the production of glycolic acid and/or glyoxylic acid. The methods of the invention involve either only one step of fermentation of the modified microorganisms of the invention or one step of fermentation of the modified microorganisms of the invention and one step of either biological or chemical conversion of the glycolic acid or of the glyoxylic acid, the microorganism of the invention over expressing a phosphoketolase gene.
LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting expression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.
Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
This invention relates to compounds, compositions, and methods useful for reducing Glycolate Oxidase (HAO1) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LDHA gene, as well as methods of inhibiting epression of LDHA, methods of inhibiting LDHA and HAO1, and methods of treating subjects that would benefit from reduction in expression of LDHA, such as subjects having an oxalate pathway-associated disease, disorder, or condition, using such dsRNA compositions.
METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GLYCOLATE OXIDASE (HAO1) BY DOUBLE-STRANDED RNA
This invention relates to compounds, compositions, and methods useful for reducing Glycolate Oxidase (HAO1) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
METHODS AND MICROORGANISMS FOR THE PRODUCTION OF GLYCOLIC ACID AND/OR GLYOXYLIC ACID
The present invention relates to methods and recombinant microorganisms useful for the production of glycolic acid and/or glyoxylic acid. The methods of the invention involve either only one step of fermentation of the modified microorganisms of the invention or one step of fermentation of the modified microorganisms of the invention and one step of either biological or chemical conversion of the glycolic acid or of the glyoxylic acid, the microorganism of the invention over expressing a phosphoketolase gene.
Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
This invention relates to compounds, compositions, and methods useful for reducing Glycolate Oxidase (HAO1) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.