Compositions and methods for treating FSHD and for identifying agents useful for the treatment of FSHD.

Cas-protein-ready tau biosensor cells, CRISPR/Cas synergistic activation mediator (SAM)-ready tau biosensor cells, and methods of making and using such cells to screen for genetic modifiers of tau seeding or aggregation are provided. Reagents and methods for sensitizing such cells to tau seeding activity or tau aggregation or for causing tau aggregation are also provided.

The present disclosure relates to the involvement of HSA-miR-6891-5p in immune and/or inflammatory disorders, as well as the use of agonists/antagonists thereof to treat the same.

Disclosed herein include systems, methods, and compositions for determining perturbations in single cells or performing integrated measurements using, for example, plasmids each comprising (i) a perturbation gRNA with a guide region targeting a chromosome sequence and (ii) barcode gRNAs each with a guide region targeting predetermined spatial region of the genome and zero, one, or more optical detection probe binding sites.

The present invention relates to a method for improving the heterologous synthesis of a polyketide by E. coli and use thereof. The yield of the polyketide heterologously synthesized by E. coli is significantly increased by attenuating the expression of seventy-two genes, such as sucC and talB, in a host strain, wherein the highest yield increase rate can reach 60% or more. Currently, erythromycin is the most clear model compound in the study on the biosynthesis of polyketids. The production strain of the present invention enables massive accumulation of 6-deoxyerythronolide (6-dEB), an erythromycin precursor, in the fermentation process, laying the foundation for the industrial production of the heterologous synthesis of erythromycin by E. coli.

A method for treating HER2 positive breast cancer in a subject in need thereof includes administering to cancer cells of the subject an agent effective to modulate the level of HER2-associated RNA in the breast cancer cells of the subject.

The present disclosure relate to a composition for inhibiting a prokaryotic expression and a use thereof and, more specifically, to a composition for inhibiting an expression of Gram-positive bacteria, which includes an sRNA comprising an sRNA-derived Hfq binding site from prokaryotes and (ii) a region that forms a complementary bond with a target gene mRNA and an Hfq from prokaryotes, a method of producing same, and a use thereof. A synthetic sRNA according to the present disclosure and a composition comprising the sRNA for inhibiting a gene expression having an advantage of being able to control single and multiple target genes at a time, can effectively reduce the expression of the target gene without the conventional gene deletion process via the synthetic sRNA that controls a gene expression so as to be useful for the production of a recombinant microorganism, and are particularly useful for inhibiting a gene expression of Gram-positive bacteria. A recombinant Corynebacterium produced by the present disclosure is a recombinant microorganism capable of mass production of high value products in an ecofriendly and reproducible manner on a bio-basis by controlling microbial metabolism flow through the synthetic sRNA. The recombinant microorganism, which is a bio-based production system developed through the sRNA is useful because of being able to replace existing fossil fuels while resolving environmental problems due to the ever-increasing use of crude oil.

The invention relates, in part, to methods and systems with which to identify guide RNAs (gRNAs) and methods and systems with which to prepare, design, and generate gRNAs and minimally repetitive arrays of gRNAs.

This invention concerns pathological angiogenesis and cancer, related treatment methods, and related compositions. Also disclosed are related diagnosis kits and methods.

BANF1, PPP2CA, and ANKLE2 were identified as genes that promote tau aggregation when disrupted. Improved tauopathy models such as cells, tissues, or animals having mutations in or inhibition of expression of BANF1 and/or PPP2CA and/or ANKLE2 are provided. Methods of using such improved tauopathy models for assessing therapeutic candidates for the treatment of a tauopathy, methods of making the improved tauopathy models, and methods of accelerating or exacerbating tau aggregation in a tauopathy model are also provided.