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This technology includes a mouse model of Pompe disease, created by targeted inactivation of the acid alpha-glucosidase gene, to test novel therapies. Pompe disease is a severe muscle disorder that affects people at any age. It is a rare genetic disease caused by a deficiency of a lysosomal enzyme acid alpha-glucosidase. The enzyme degrades glycogen to glucose in the lysosomes. The deficiency leads to accumulation of glycogen in multiple organs, but cardiac and skeletal muscles are most severely affected.

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 includes the creation of DLX3-floxed mice, specifically designed for conditional deletion of the DLX3 gene via Cre-mediated recombination. This innovative approach aims to develop mouse models for studying ectodermal dysplasia disorders. Ectodermal dysplasias are a diverse group of genetic conditions affecting the development of ectodermal structures, including hair, teeth, and bones. The DLX3f/f mice are particularly valuable for modeling specific disorders such as Tricho-dento-osseous syndrome (TDO), Amelogenesis Imperfecta (AI), and Dentinogenesis Imperfecta (DI).

This technology includes K14creDLX3 conditional knockout (cKO) mice which will be used to study ectodermal dysplasia disorders such as Amelogenesis Imperfecta, and to study molecular mechanisms of DLX3 regulation in skin and ectodermal appendages. DLX3 is expressed in the epidermis, hair matrix cells in the hair follicle and in the mesenchymal and epithelial compartment of the tooth during embryonic development. To determine the transcriptional network dependent on DLX3-function, we will generate and analyze an epithelial-specific conditional knockout of DLX3.

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 an innovative method for differentiating astrocytes from neural stem cells (NSCs). The process involves using Life Technologies StemPro embryonic stem cell serum-free medium to initially guide NSCs towards a neuronal lineage. Over a period of 28-35 days, as the cells are continually passaged, neurons gradually die off, leading to the proliferation of astrocytes. By the end of this differentiation protocol, approximately 70% of the cells exhibit markers characteristic of mature astrocytes, specifically GFAP.

This technology includes two safe harbor gene targeting donors, specifically designed for applications in the study of induced pluripotent stem cells (iPSC). These include the pAAVS1D-CMV.RFP-EF1a.copGFPpuro and pAAVS1-iCLHN donors. A key feature of these donors is their ability to integrate various transgenes into specific loci through homologous recombination, facilitated by sequences homologous to safe harbor loci. When paired with TALENs targeting these loci, these plasmids enable precise and efficient genome engineering in human cells.

This technology includes apoE-apoCII chimeric peptides that possess the ability to lower the triglyceride level both in vitro and in vivo. These peptides can be used for treating hypertriglyceridemia and alleviating other diseases and conditions associated with increased triglycerides.

Pre-exposure prophylaxis (PrEP) is a critical component in the fight against HIV but is only effective if persons prescribed PrEP are adhering to the regimens to maintain appropriate drug levels. As PrEP regimens have moved from daily pills to longer lasting injections, the ability to quickly measure and monitor the circulating drug levels of PrEP drugs has increased importance.