Abstract: Provided herein are methods and tools for targeting detecting (e.g., imaging) extrachromosomal DNA, for example, in cancer cells.

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 disclosure provides, in some aspects, humanized immunodeficient mouse models that support long-term engraftment and function of human T cells, natural killer cells, and myeloid cells, without the need for conditioning.

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 disclosure relates to methods and compositions that regulate splicing in NASP transcripts.

Abstract: Clonal hematopoiesis is an age-related condition caused by somatic mutations that give a hematopoietic stem cell a clonal selective advantage. While clonal hematopoiesis is a benign condition, individuals affected by it have an increased risk of developing blood cancers, such as acute myeloid leukemia (AML). The present disclosure provides, in some aspects, methods for inhibiting clonal hematopoiesis using a senolytic agent to target senescence of bone marrow stromal cells.

Abstract: Provided are methods of targeting patient-specific oncogenes in extrachromosomal DNA (ecDNA) to treat glioma in a human. The present methods include identifying a drug that targets against an oncogene present in ecDNA of a human suffering from glioma, such as glioblastoma. The identified oncogenes present in ecDNA include MET, MET/CAPZA2, MDM2, CDK4, SOX2, PIK3CA, MECOM, PDGFRA, EGFR, MYCN, MYC, TERT, SMARCA4, RP56, FBXW7, CDK6, CCND2, ERBB2, BRCA1, and BAP1. The present methods include identifying a drug targeted against the ecDNA oncogene, which drug inhibits the function of the identified oncogene, so as to inhibit tumor growth or progression of the glioma in the human. Also provided are PDX mouse models to further identify and/or confirm patient-specific drugs that target the identified oncogene(s) present in ecDNA. Also provided are methods of diagnosing gliomas or recurrent gliomas and methods of screening or monitoring for recurrence of gliomas.

Abstract: Provided herein are methods and compositions for the treatment or prevention of certain disorders and conditions, for example, addiction using an agent to modulate 5-hydroxytryptamine 1D receptor (HTR1D) activity and/or expression. Also provided are methods for screening a candidate to determine if the candidate is suitable for the therapies disclosed herein.

Abstract: A method of identifying anti-tumor activity of a test substance is provided along with a genetically-modified, immunodeficient mouse and methods of use, wherein the genetically-modified, immunodeficient mouse enables in vivo investigation of the interactions between the human immune system and human cancer.

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 are humanized mouse models generated using T cell-negative fractions or peripheral blood mononuclear cells obtained from T cell-negative fractions, and methods of using the mouse models to assess the efficacy and/or side effects of a therapeutic agent. Immune cell therapies require a large number of cells. Most commonly, the cells are collected using a process referred to as apheresis. Apheresis collection of the mononuclear cell (MNC) layer has been shown to be a safe and efficient method of collecting the large number of T cells.

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: The present disclosure provides wild-derived mouse models that comprise a nucleic acid encoding a human amyloid precursor protein (APP), a nucleic acid encoding a mutated human presenilin (1) protein (PSEN1), and in some embodiments, a human apolipoprotein E (APOE), or human amyloid beta and human tau. These mouse models are useful, for example, for Alzheimer's disease studies.

Abstract: Provided herein, in some aspects, is a NOD.Cg-Prkdcscid Il2rgtm1Wj1/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: An anchoring device may include a body including a cavity configured to receive a data logger disposed therein. An anchoring device may include an anchoring portion attached to the body. An anchoring device may include one or more through holes formed in the anchoring portion, where the one or more through holes are configured to accept a suture passing therethrough to attach the anchoring device to tissue of a subject.

Abstract: Provided herein, in some embodiments, are methods and compositions for the treatment of Charcot-Marie-Tooth disease.

Abstract: Provided herein are humanized immunodeficient mouse comprising human myeloid cells and human lymphoid cells, wherein the human lymphoid cells comprise human B cells that produce circulating immunoglobulin (Ig), for example, physiological levels of human IgG, and wherein the mouse expresses a detectable level of human FLT3L protein and does not express a detectable level of mouse FLT3 protein.

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: A method of identifying anti-tumor activity of a test substance is provided along with a genetically-modified, immunodeficient mouse and methods of use, wherein the genetically-modified, immunodeficient mouse enables in vivo investigation of the interactions between the human immune system and human cancer.

Abstract: Techniques for classifying, using a deep learning model, histopathological whole slide images (WSIs) as comprising images of cancerous or non-cancerous tissue and/or as comprising images of cancerous tissue having a genetic mutation or not having a genetic mutation are described herein. The techniques include at least one processor configured to instantiate a container-based processing architecture to train and/or use the deep learning model to process and classify at least one WSI. In some embodiments, a treatment may be selected and administered based on a classification result obtained from the deep learning model.