This technology includes mouse models that express versions of mouse cryopyrin protein containing mutations associated with human CAPS disease. We engineered mutations associated with three specific CAPS phenotypes (familial cold autoinflammatory syndrome (FCAS); Muckle-Wells syndrome (MWS); and neonatal onset multisystem inflammatory disease (NOMID)) into the mouse cryopyrin gene (called Nlrp3) to examine the roles of IL-1 β and related cytokines, and better characterize inflammasome functions.

This technology includes the use of genetic markers to predict the response of patients, particularly children with systemic juvenile idiopathic arthritis (sJiA), to anakinra treatment. Anakinra is a human recombinant IL-1 RA used in treating sJiA, a severe childhood inflammatory disease where early and effective treatment is essential for better long-term outcomes. Through the analysis of 38 children with sJiA treated with anakinra, specific sJiA-associated SNPs (single nucleotide polymorphisms) were identified as predictors of therapeutic failure, with a significant odds ratio of 17.3.

This technology includes the development of a mouse line capable of producing single-chain antibodies (nanobodies). Nanobodies, identified initially from Camelidae (including llamas and camels) but also found in cartilaginous fish, consist of a single variable heavy chain domain (VHH) that binds to specific epitopes. Nanobodies have equivalent binding specificity to antigens as antibodies but are more heat- and detergent-stable.

This technology involves the utilization of highly effective nanobodies, specifically camelid antibodies, derived from immunized llamas to neutralize SARS-CoV-2. Additionally, it employs a unique mouse model, called a "nanomouse," that is engineered to express antibody genes from camels, alpacas, and dromedaries. These nanobodies offer significant advantages over traditional human and mouse antibodies due to their smaller size, which allows them to effectively target and bind to specific areas on antigens.

This technology includes mouse models of TL 1A diseases, such as inflammatory bowel disease and rheumatoid arthritis, to be used as a platform for studying therapeutic agents. The TNF family cytokine TL 1A co-stimulates T-cells through Its receptor and is required for autoimmune pathology driven by diverse T-cell subsets. Blocking TL 1A in mouse models of these diseases is efficacious blocking TL 1A may be useful for therapy of diseases in which TL 1A plays a pathogenic role.

This technology involves an innovative method for differentiating neural stem cells (NSCs) into neurons, primarily for use in basic science research and in developing therapies for brain and spinal cord disorders. Existing methods for generating neurons from NSCs typically result in high efficiency but low survival rates, especially when neurons are dissociated and regrown. This new method utilizes Life Technologies StemPro embryonic stem cell serum-free medium, which significantly enhances differentiation efficiency into neurons with minimal cell death.

This technology includes a neural stem cell (NSC) line derived from a Niemann Pick C (NPC) patient, aimed at advancing research and drug development for NPC, an inherited neurodegenerative disorder characterized by the accumulation of cholesterol in neurons. The NSCs, which serve as a crucial intermediate cell type, can be differentiated into any neuronal or glial cell of the brain or spinal cord under appropriate culture conditions. These cells originate from fibroblasts reprogrammed into induced pluripotent stem cells.

This technology involves neural stem cells (NSCs) derived from pluripotent stem cells (PSCs) that can differentiate into neurons and glia. The key feature of this technology is the CY2 EEF1A1 GFP iPSC line, which includes a green fluorescent protein (GFP) expressed under the EEF1A1 promoter, leading to its ubiquitous expression in cells. This characteristic makes the NSCs and the neural cells differentiated from this line exhibit green fluorescence. Such cells, when transplanted into animal models like mice and rats, can be easily tracked due to their fluorescence.

This technology includes a novel plasmid design for cell immortalization. It uniquely combines the conditional activation of human telomerase and c-myc genes through cumate addition, a method distinct from traditional immortalization techniques which commonly use SV40 T-antigen, telomerase, or c-myc. This plasmid also includes a GFP reporter and a puromycin resistance gene, enhancing the efficiency of the immortalization process.

This technology includes the use of engineered human induced pluripotent stem cells (iPSCs) for various applications such as studying cell differentiation, drug screening, and gene transfer therapy. It employs gene targeting donors flanked by DNA sequences compatible with endogenous loci to integrate transgenes through homologous recombination. A key aspect is the flexible gene targeting donor design, used in conjunction with safe harbor transcription activator-like effector nucleases (TALENs).