Fibroblast Cell Lines (with L444P/RecNci1 Genotype) for the Screening of Small Molecules for Gaucher Disease Treatment

This technology includes two human fibroblast cell lines to be used to study the defects in GBA1 gene and protein and to screen small molecules for involvement in Gaucher disease. Glucocerebrosidase (GBA1 or GCase or beta-glucosidase) is a lysosomal enzyme, responsible for breakdown of a fatty material called glucocerebroside (or glucosyl ceramide). Deficiency or malfunction of GBA1 leads to the accumulation of insoluble glucocerebrosides in tissues, which is a major symptom of Gaucher disease.

Systemic CRISPR Therapy for the Treatment of Inherited Diseases

This technology includes novel systemic adeno-associated virus (AAV)-mediated CRISPR gene therapy technology. While some diseases (e.g., retinal diseases) can be treated through local gene transfer, many diseases such as Duchenne Muscular Dystrophy (DMD) require systemic therapy. The CRISPR technology has two components, the Cas9 endonuclease, and the gRNA. To explore systemic CRISPR therapy, we co-delivered the AAV.Cas9 and AAV.gRNA vector to mdx mice, a mouse DMD model. Direct delivery to muscle yielded efficient gene correction.

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.

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).

CXCR4 Reduction Leads to Enhancement of Engraftment of Hematopoietic Stem Cells

Methods of enhancing engraftment of donor hematopoietic stem cells (HSCs) by reducing expression or activity of CXCR4 in HSCs is described. HSC are the only cells in the bone marrow that are both pluripotent and long lived. Bone marrow transplantation (BMT) using HSC is an increasingly common medical therapy for severe hematologic cancers and primary hematologic immunodeficiencies. However, for significant HSC engraftment to occur there must usually be pre-transplant conditioning with either irradiation or chemotherapy or both.

LRRK2 Inhibitors: Novel Treatment for Intestinal Bowel Disorders

Use of Leucine Rich Repeat Kinase 2 (LRRK2) inhibitors for the treatment of Intestinal Bowel Disorders (IBD) is disclosed. IBD is a broad term that describes conditions with chronic or recurring immune response and inflammation of the gastrointestinal tract. Crohn's disease and ulcerative colitis, two common forms of idiopathic IBD, are chronic, relapsing inflammatory disorders of the gastrointestinal tract.

Potency Assay for Membrane Transporter Protein-based Drugs Acting on Antioxidant, Redox, and Apoptosis Response Pathways

Description of Technology:

This technology includes a method of analyzing the potency of membrane transporter protein-based drugs acting on intracellular antioxidant and redox response pathways (and associated apoptosis pathways), wherein the drug delivery and activity is lipid associated. The present invention is a cell-based bioassay for measuring the bioactivity of drug substance and formulated drug product by determining the drug's dose-dependent inhibitory effects on 4 hydroxynonenal (4-HNE)-induced antioxidant response element (ARE) activity.

Subscribe to Dental