KCNN4 Knockout Mice for Mechanistic Research

This technology includes a transgenic allele for a mouse knockout model for the KCNN4 gene. Secretion of fluids from these salivary glands requires the coordination of multiple water and ion channel proteins. Notably, the majority of these channels have been shown to be up-regulated by increased calcium concentrations. The relevant calcium-activated potassium channels are split into the small, intermediate, and large conductance channels (called the SK, IK, and BK channels). The KCNN4 gene plays a part in the IK and BK channels.

Rescue of AAV Production by shRNA Co-transfection

Recombinant adeno-associated virus (rAAV) vectors are proving to be a valid, safe and efficient gene transfer system for clinical applications. As most vectors utilize constitutive promoters, this results in transgene expression in the producer cell. Some of these transgene products can induce proapoptotic, cytostatic or other unknown effects that interfere with producer cell function. Therefore, this reduces the viral vector yield and is a major limitation when trying to characterize poorly described genes.

Alpha-galactosidase-A Knockout Mouse Model for Studying Fabry Disease

This technology includes an alpha-galactosidase-A knockout mouse model that can be used to study Fabry disease, an X-linked lysosomal storage disorder. Alpha-galactosidase-A is a crucial enzyme responsible for the breakdown of glycolipids, particularly globotriaosylceramide (Gb3), within lysosomes. In Fabry disease, a rare and inherited lysosomal storage disorder, mutations in the GLA gene lead to deficient or non-functional alpha-galactosidase-A enzyme activity.

Method To Generate Chondrocytes from Human Induced Pluripotent Stem Cells (hIPSCs) and their use in Repairing Human Injury and Degenerative Diseases

This technology includes a method for differentiating human induced pluripotent stem cells (hiPSCs) into stable chondrocytes, capable of producing cartilage, and their use in cartilage repair in human injury and degenerative diseases. In suspension culture, hiPSC aggregates demonstrate gene and protein expression patterns similar to articular cartilage.

Advancing VZV Antibody Detection: A High-Throughput LIPS Assay for Varicella Vaccine Recipients

The technology described is a sophisticated and high-throughput luciferase immunoprecipitation system (LIPS) assay designed to detect antibodies specific to Varicella-zoster virus (VZV) glycoprotein E (gE). By transfecting cells with VZV protein-Renilla luciferase fusion protein constructs and subsequently performing immunoprecipitations with protein A/G beads, this innovative assay enables the quantitative measurement of VZV gE antibody levels in blood serum samples.

Diagnostic Assays and Methods of Use for Detection of Filarial Infection

The effort targeting the mosquito borne neglected tropical disease lymphatic filariasis for elimination through mass drug administration by 2020 will require accurate, cost effective methods for detecting early infections. The World Health Organization-recommended immunochromatographic test detects adult Wuchereria bancrofti (Wb) antigen in blood, but shows variable efficacy due to the complex life cycle of the parasites and cross reactivity with other organisms. This variability may hinder effective lymphatic filariasis elimination efforts.

Treatment of Periodontal Disease via ENPPI Inhibition

This technology focuses on enhancing cementum production, a key component in treating periodontal regression. The method involves inhibiting ectonucleotide pyrophosphatase phosphodiesterases (ENPP1), enzymes that play a significant role in mineralization processes. Pyrophosphate (PPi) is known to impede the growth of hydroxyapatite crystals, essential for mineralization. ENPP1 catalyzes the hydrolysis of ATP, generating PPi, which then hinders mineralization.

Postnatal Stem Cells and Uses Thereof

Many individuals with ongoing and severe dental problems are faced with the prospect of permanent tooth loss. Examples of such dental problems include: dentinal degradation due to chronic dental disease (caries or periodontal); mouth injury; or through surgical removal, such as with tumors associated with the jaw. For many, a technology that offers a possible alternative to artificial dentures by designing and transplanting a set of living teeth fashioned from an individual's own pulp cells would greatly improve their quality of life.

Rabbit Antisera to Various Matrix, Matricellular, and Other Secreted Proteins

The extracellular matrix (ECM) is composed of a group of proteins that regulate many cellular functions, such as cell shape, adhesion, migration, proliferation, and differentiation. Deregulation of ECM protein production or function contributes to many pathological conditions, including asthma, chronic obstructive pulmonary disease, arthrosclerosis, and cancer. Scientists at the NIH have developed antisera against various ECM components such as proteoglycan, sialoprotein, collagen, etc.. These antisera can be used as research tools to study the biology of extracellular matrix molecules.

In-vivo System to Interrogate the Functions of Mucous Membranes and Identify Mucin/Glycan Mimetics and JAK/STAT Inhibitors for the Treatment of Diseases of the Oral Cavity and Digestive Tract

This technology includes a Drosophila mutant strain that can be used as an in vivo model for diseases of the oral cavity and digestive tract (Sjogren's syndrome, colitis, colon cancer, inflammatory bowel disease), where the mucous membrane is disrupted or non-functional. This mutant lacks a mucous membrane and displays epithelial cell damage, uncontrolled cell proliferation and the up-regulation of conserved signaling pathways (JAK/STAT).
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