The present disclosure discloses a methicillin-resistant staphylococcus aureus mutant strain and use thereof, and belongs to the field of molecular biology and microorganisms. The methicillin-resistant staphylococcus aureus mutant strain disclosed by the present disclosure has a relatively low exopolysaccharide synthesis ability and a relatively low biofilm metabolism ability, but it is sensitive to an antibiotic cefoxitin. The mutant strain can be used for treating a related disease caused by methicillin-resistant staphylococcus aureus infection through an endogenous ecological treatment strategy. The present disclosure provides a new idea for treating the disease.

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.

The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.

An RNA molecule is disclosed having a sequence having at least 80% sequence identity to SEQ ID NO: 2. The invention is also directed to an RNA molecule obtainable by transcription of a DNA sequence having at least 80% sequence identity to SEQ ID NO: 1 for use as a medicament and to pharmaceutical compositions comprising such an RNA molecule.

The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.

The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier and method for treating diseases caused by PCSK9 gene expression.

Provided is a composition comprising at least one selected fragment of DNA exogenous to a targeted organism and a carrier, wherein said DNA fragment has at least 50% sequence homology to a DNA sequence of said targeted organism and wherein said fragment comprises no more than 5 unprotected specific endonuclease recognition sites of said targeted organism. Further provided are methods of use of the composition. Further provided are methods for producing the composition.

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A) in a cell or subject, and in certain instances increasing the expression of paternal UBE3A and the amount of UBE3A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurogenetic disorder. Such symptoms and hallmarks include developmental delays, ataxia, speech impairment, sleep problems, seizures, and EEG abnormalities. Such neurogenetic disorders include Angelman Syndrome.