Abstract: A genetically-modified, immunodeficient mouse is provided along with methods of use, wherein the mouse includes (a) a nucleotide sequence encoding human stem cell factor (hSCF); (b) a nucleotide sequence encoding human granulocyte-macrophage colony-stimulating factor (hGM-CSF); (c) a nucleotide sequence encoding human interleukin-3 (hIL-3); and (d) a nucleotide sequence encoding human colony-stimulating factor 1 (hCSF1), wherein each of the nucleotide sequences is operably linked to a promoter, and wherein the genetically-modified, immunodeficient mouse expresses hSCF, hGM-CSF, hIL-3, and hCSF1, wherein the genetically-modified, immunodeficient mouse allows engraftment of human hematopoietic stem cells along with engraftment of human-patient derived tumor xenografts and/or human tumor cell lines to enable in vivo investigation of the interactions between the human immune system and human cancer.

Abstract: One or more embodiments of the present invention include an immunodeficient mouse genetically modified to include a gene encoding a PiZ variant (Glu342Lys) of human ?-1 antitrypsin (AAT), wherein the mouse expresses the PiZ variant of human ?-1 antitrypsin (Z-AAT), and has a reduced number of mouse hepatocytes compared to an immunodeficient mouse of the same type which does not express Z-AAT. The immunodeficient mouse genetically modified to include a gene encoding a PiZ variant of human AAT can be a genetically modified NSG, NRG or NOG mouse. The immunodeficient mouse may further include xenogeneic hepatocytes, such as human hepatocytes. Putative treatments of human liver disease can be assessed in mice provided according to aspects of the present disclosure.

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: Provided herein are, inter alia, compositions and methods for the delivery of enhanced demethylation activity to target DNA sequences in a mammalian cell. The compositions and methods are, useful for activity modulation of a targeted gene, or to create a gene regulatory network.

Abstract: A method for treating, preventing and/or reducing neurodegeneration in subjects with neurodegenerative disease, such as those neurodegenerative diseases that affect the eye, including glaucoma, using radiation, such as gamma radiation or X-ray radiation, either alone or together with a bone marrow transfer treatment. The method includes irradiating a targeted area of an animal, such as the eye region, with radiation, either alone or followed by injection with T-cell depleted bone marrow cells. Also a method for screening and/or selecting agents and/or treatment methods for inhibiting, treating and/or reducing neurodegeneration, particularly the neurodegeneration of the eye that occurs as a consequence of glaucoma.

Abstract: Provided herein, in some embodiments, are methods for classifying the tandem duplicator phenotype of a tumor.

Abstract: Provided herein, in some embodiments, are methods comprising culturing human brain tissue spheroids on a three-dimensional scaffold in culture media comprising vascular endothelial growth factor (VEGF), and producing a population of cells, wherein cells of the population are positive for nestin, glial fibrillary acidic protein (GFAP), and/or vimentin.

Abstract: A genetically modified immunodeficient non-human animal whose genome includes a genetic modification that renders the non-human animal deficient in macrophages and/or macrophage anti-human red blood cell activity so as to prolong the survival of human red blood cells when administered into said non-human animal is provided according to aspects of the present invention. Methods of assaying effects of putative therapeutic agents in such a genetically modified immunodeficient non-human animal are provided by the present invention.

Abstract: The invention described herein provide a method to quantitatively determine the extent of tandem duplications (TDs) in certain cancer, and the use of the novel scoring metric to determine whether the cancers exhibiting tandem duplicator phenotype (TDP) and their increased susceptibility to platinum-based chemotherapeutic agents.

Abstract: The present disclosure provides, in some aspects, a humanized mouse (NSG™-SGM3), engrafted with CD34+ hematopoietic progenitor cells and human metastatic melanoma cells, which surprisingly promotes secondary metastatic colonization, modeling the interplay between human tumors and the human immune system, and thus enabling mechanistic and pre-clinical studies for the development of novel treatment strategies targeting human-specific molecular pathways controlling melanoma dissemination.

Abstract: Presently disclosed are methods of treating cancer comprising administering a MEK inhibitor in combination with a proteasome inhibitor. In some embodiment, the cancer is a solid tumor. In some instances, the cancer has at least one mutation chosen from a NF1, RAS (including N-, K-, and H-RAS), RAF (including A-, B-, and C-RAF), and MEK (including MEK1 and MEK2) mutation. In some embodiments, the cancer is resistant to treatment with at least one of a proteasome inhibitor or a MEK inhibitor. In some embodiments, the combination therapy produces a synergistic effect.

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 described herein relates to methods of screening for pro-inflammatory genes and anti-inflammatory genes which may be useful for treating an inflammatory disease, disorder, or otherwise abnormal condition, such as an inflammatory lung disease. The identified pro-inflammatory genes and anti-inflammatory genes may be used to produce pharmaceutical compositions for use in treating the inflammatory disease, disorder, or otherwise abnormal condition.

Abstract: Described herein are immunodeficient non-human animals lacking expression of toll-like receptor 4 (TLR4) by endogenous autogeneic innate immune cells, as well as methods and compositions for engraftment of xenogeneic hematopoietic stem cells in the immunodeficient non-human animal lacking expression of toll-like receptor 4 (TLR4), thereby creating an innate immune system in the animal derived from the xenogeneic hematopoietic stem cells. Further described are immunodeficient mice lacking expression of toll-like receptor 4 by endogenous autogeneic innate immune cells, as well as methods and compositions for engraftment of xenogeneic hematopoietic stem cells in the immunodeficient mouse lacking expression of toll-like receptor 4, thereby creating an innate immune system in the animal derived from the xenogeneic hematopoietic stem 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: A biomaterial handling device is described that will provide for functionality for performing both IVF procedures and washing techniques in a single device. Disclosed embodiments provide increased protection to biomaterial samples during processing and handling. Embodiments of the invention reduce labor intensive processes for both IVF and washing treatments and address reduced risks of contamination of biological samples by providing an increasingly sterile environment.

Abstract: The invention described herein provides methods and systems for comprehensive genomic analysis that enables the detection of a broad range of genomic variations, including single nucleotide polymorphisms (SNPs), small insertions or deletions (indels), Tandem Base Mutations (TBM), copy number variations (CNVs), structural variations (SVs), and combination thereof, in a single assay. The invention can be used, for example, to analyze the complicated underlying genomic defects in diseases and conditions such as Autism spectrum disorders (ASD), cancers, Alzheimer's disease, and other neurological disorders.

Abstract: The invention described herein relates to methods of screening for pro-inflammatory genes and anti-inflammatory genes which may be useful for treating an inflammatory disease, disorder, or otherwise abnormal condition, such as an inflammatory lung disease. The identified pro-inflammatory genes and anti-inflammatory genes may be used to produce pharmaceutical compositions for use in treating the inflammatory disease, disorder, or otherwise abnormal condition.

Abstract: The invention described herein provides reagents (e.g., kits), compositions, and methods for carrying out an unbiased genome-wide strategy to identify the functional targets for all ncRNAs.

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.