Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Abstract: A key-value store and a file system are integrated together to provide improved operations. The key-value store can include a log engine, a hash engine, a sorting engine, and a garbage collection manager. The features of the key-value store can be configured to reduce the number of I/O operations involving the file system, thereby improving read efficiency, reducing write latency, and reducing write amplification issues inherent in the combined key-value store and file system.

Abstract: Provided are a method, a device and electronic equipment for detecting a stability of a generator set in a power system. The method includes following steps: determining a target matrix by using Liapunov's direct method based on a state space matrix corresponding to power management unit (PMU) data; obtaining a state deviation corresponding to the PMU data and an oscillation curve corresponding to the state deviation in a case of determining the power system being in a stable state according to the target matrix; determining extreme point values corresponding to the oscillation curve at each oscillation; and evaluating a working state of the generator set in the power system according to the extreme point values.

Abstract: Described are compounds and methods useful in the treatment of Fuchs' Endothelial Corneal Dystrophy (FECD).

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Abstract: Described are compounds and methods useful in the treatment of Fuchs' Endothelial Corneal Dystrophy (FECD).

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Abstract: Molecular biomarkers relating to Fuchs' endothelial corneal dystrophy (FECD), glaucoma, and other degenerative ocular diseases are provided, as well as methods for using such biomarkers methods of treatment. These biomarkers may be used to monitor the progression of FECD, glaucoma, or other degenerative ocular diseases. Furthermore, these biomarkers may be used to monitor the treatment of FECD, glaucoma, or other degenerative ocular diseases.

Abstract: Described are compounds and methods useful in the treatment of Fuchs' Endothelial Corneal Dystrophy (FECD).

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Provided herein are a double-stranded oligonucleotides of 13 to 22 nucleobases in length targeting a GGGGCC expanded repeat region in an intron of C9orf72, comprises (a) 3-5 central mismatches (within bases 9-14) within a target sequence comprising the expanded repeat sequence, or (h) 3-5 mismatches outside of the seed sequence (bases 2-8 within the guide strand complementary to the expanded repeat sequence).

Abstract: Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Provided herein are a double-stranded oligonucleotides of 13 to 22 nucleobases in length targeting a GGGGCC expanded repeat region in an intron of C9orf72, comprises (a) 3-5 central mismatches (within bases 9-14) within a target sequence comprising the expanded repeat sequence, or (h) 3-5 mismatches outside of the seed sequence (bases 2-8 within the guide strand complementary to the expanded repeat sequence).

Abstract: The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntingtin Disease Protein and Ataxin-3 using double-stranded RNAs and nucleic acid analogs. Chemically-modified RNAs having at least one mismatch as compared to the target CAG repeat sequence are specifically contemplated.

Abstract: Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.

Abstract: The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntington using nucleic acid analogs. Peptide nucleic acids and locked nucleic acids are particularly useful analogs.

Abstract: The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntington using nucleic acid analogs. Peptide nucleic acids and locked nucleic acids are particularly useful analogs.

Abstract: The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntingtin using nucleic acid analogs. Peptide nucleic acids and locked nucleic acids are particularly useful analogs.

Abstract: The present invention relates to the selective modulation of pre-mRNA splicing, in particular, for that involving alternative splicing in disease-related proteins such as those involved in Duschenne's Muscular Dystropy and Spinal Muscular Atrophy.