Abstract: The invention described herein provides compositions and reagents for assembling a tripartite complex at a specific location of a target DNA. The invention also provides methods for using the complex to, for example, label a specific genomic locus, to regulate the expression of a target gene, or to create a gene regulatory network.

Abstract: Systems and methods for continuous monitoring of the behavior of animals, such as small rodents, are provided. Monitoring can include video, audio, and other sensor modalities. In one embodiment, the system can include cameras, arena design, environmental sensors, and ultrasonic sensors. The system uniquely provides a continuous long-term monitoring system suitable for mouse behavioral study. Further provided is a neural network based tracker configured for use with video data acquired by the monitoring system. Three different neural network architectures have been tested to determine their performance on genetically diverse mice under varying environmental conditions. It has been observed that that an encoder-decoder segmentation neural network achieves high accuracy and speed with minimal training data.

Abstract: In certain embodiments, this present invention provides antibodies and Fc fusion proteins with enhanced pharmacokinetics, such as biotinylated antibodies or biotinylated Fc fusion polypeptides.

Abstract: The invention relates to the use of a pharmaceutical composition containing nicotinamide (NAM) and/or pyruvate as a neuroprotective medicament or gene therapy in the treatment of neurodegenerative disorders, in particular axon degeneration of neuronal tissue in ocular-related neurodegeneration diseases including glaucoma.

Abstract: The invention, in part, encompasses methods to identify extrachromosomal circular DNA (ecDNA) and methods to identify and assess interactions between ecDNA and oncogene transcription.

Abstract: Provided herein are humanized mouse models and methods for determining whether administration of engineered immune cell therapies likely elicit cytokine release syndrome and/or determining the efficacy of an anti-disease therapy. Further, the models provided herein may be used to test the efficacy of different anti-CRS therapies.

Abstract: The present disclosure relates to methods and compositions utilizing upper-airway microbiota for diagnosing individuals at risk for asthma exacerbations. Further provided herein are probiotic compositions and methods for treating asthma by administering microorganisms that are associated with decreased risk of asthma exacerbations.

Abstract: The present invention relates to the use of lipid/fat droplet deposits in retinas of a human subject as a biomarker for diagnosing, prognosing, and/or monitoring retinal neurodegeneration with or without an elevated intraocular pressure in glaucoma.

Abstract: Systems and methods for continuous monitoring of the behavior of animals, such as small rodents, are provided. Monitoring can include video, audio, and other sensor modalities. In one embodiment, the system can include cameras, arena design, environmental sensors, and ultrasonic sensors. The system uniquely provides a continuous long-term monitoring system suitable for mouse behavioral study. Further provided is a neural network based tracker configured for use with video data acquired by the monitoring system. 3 different neural network architectures have been tested to determine their performance on genetically diverse mice under varying environmental conditions. It has been observed that that an encoder-decoder segmentation neural network achieves high accuracy and speed with minimal training data.

Abstract: YAP1 inhibitors and methods of treating a subject with a cancer that include administering a YAP1 inhibitor are disclosed. Also disclosed are methods for determining responsiveness to a YAP1 inhibitor in a subject, methods for diagnosing a tumor in a subject including determining the level of YAP1 expression, compositions that include YAP1 inhibitors, and methods of using those compositions.

Abstract: Provided herein, in some aspects, is a NOD.Cg-Prkdcscid Il2rgtm1wjl/SzJ (NOD scid gamma or NSG™) mouse comprising a nucleic acid encoding human FLT3L and an inactivated mouse Flt3 allele, methods of producing the mouse, and methods of using the mouse.

Abstract: The invention described herein provides non-HLA matched humanized mouse model (e.g., NSG mouse model) with patient-derived xenograft (PDX), as well as methods of making and using the same.

Abstract: The invention described herein provides non-HLA matched humanized mouse model (e.g., NSG mouse model) with patient-derived xenograft (PDX), as well as methods of making and using the same.

Abstract: The present invention generally relates to uses of glial cell line-derived growth factor (GDNF) in cutaneous wound healing and hair growth. Methods of effecting hair growth and/or wound healing which feature administration of GDNF, or a biologically active fragment thereof, to subjects, e.g., human subject, are disclosed herein. The invention relates also to formulations and kits for achieving the indicated pharmaceutical advantages.

Abstract: Techniques for detecting copy number variations (CNVs) in a genetic sequence, diagnosing disorders caused by CNVs, and treating disorders caused by CNVs are presented. The techniques include using a processor to perform steps of: scanning the genetic sequence to identify genetic regions corresponding to at least one autosomal chromosome, dividing the genetic sequence into bins, calculating a CNV status for each bin of the plurality of bins, and filtering the CNV statuses to identify at least one CNV in the genetic sequence.

Abstract: The invention described herein provides non-HLA matched humanized mouse model (e.g., NSG mouse model) with patient-derived xenograft (PDX), as well as methods of making and using the same.

Abstract: Humanized mouse models and methods are provided for determining whether administration of an immunomodulatory drug likely elicits a severe cytokine release syndrome in a human. Humanized mouse models and methods are also provided for determining the immunotoxicity in a human of a drug candidate or of drug combinations.

Abstract: Provided herein, in some embodiments, are methods and compositions for the treatment of Charcot-Marie-Tooth disease, comprising an inhibitor of a GCN2 pathway component.

Abstract: Provided herein, in some aspects, are compositions and methods for artificially modulating alternative splicing, for example, inducing exon inclusion and/or exon exclusion events. In some embodiments, a catalytically inactive programmable nuclease, such as dCasRx, is fused to an RNA-binding protein (or fragment or isoform thereof) and, when guided to a target of interest by a specific guide RNA (gRNA), can regulate alternative splicing in eukaryotic cells.

Abstract: Methods and apparatus for providing clinical decision support. The method comprises receiving phenotype information for a patient, determining a likelihood ratio for each of the phenotype features included in the received phenotype information with respect to each of a plurality of diseases, determining, based on the likelihood ratio for each of the phenotype features, a composite likelihood ratio for each of the plurality of diseases, ranking the plurality of diseases based, at least in part, on the determined composite likelihood ratios, and displaying at least some of the ranked plurality of diseases.