Glucocerebrosidase Activators as a Treatment for Gaucher Disease

This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Gaucher disease is a rare disease affecting 1 in 40,000 babies born. Ashkenazi Jews of eastern European descent (about 1 in 800 live births) are at particular risk of carrying this genetic defect. It is caused by inherited genetic mutations in the gene that encodes GCase, which result in reduced activity of the enzyme.

Levonorgestrel Butanoate Formulation and Methods Relating Thereto

This invention is a potential subcutaneous or intramuscular progestin-only, injectable contraceptive for women. Forty-five percent (45%) of pregnancies in the United States are unintended. In this group, one-third of reproductive age women are obese – increasing the risk of diabetes, hypertension and venous thromboembolism (VTE). All these are conditions for which most hormonal methods are contraindicated. Thus, additional safe and effective injectable contraceptive options are needed.

Monomeric and Oligomeric Compounds as Contraceptives and Endocrine Therapeutics

The options for male contraceptives are limited. Research is ongoing to develop a male contraceptive based on hormonal activity. Testosterone is one of the hormones necessary in producing sperm.  Testosterone is absolutely required as a hormone for male fertility. Derivatives of testosterone for male contraceptives currently in clinical trials are associated with estrogenic deficiency. This deficiency can cause several issues including, but not limited to, bone density loss, risk of obesity, cardiovascular disease, and/or ineffective carbohydrate or lipid metabolism. 

Mouse Embryo Culture Chamber and Imaging System and Methods of Use

The culture of mouse embryos ex utero and continuous monitoring and imaging of embryos as they develop have applications in drug testing, genetic studies, and basic research on embryonic development. However, the embryo culture systems currently available for post-implantation embryos include rolling bottle culture systems, which do not permit imaging of the developing embryos and do not support the long-term survival and development of embryos ex utero.

New Insect Sf9-ET Cell Line for Determining Baculovirus Titers

The baculovirus-based protein expression system has gained increased prominence as a method for expressing recombinant proteins that are used in a wide range of biomedical applications. An important step in the use of this system is the ability to determine the virus infectious titer, i.e., the number of active baculovirus particles produced during an infection of the insect host cell.

A peptide hydrogel for use in vascular anastomosis

In collaboration with surgery specialists from Johns Hopkins University, researchers at the National Cancer Institute (NCI) developed novel hydrogel compositions and methods of using them in the microsurgical suturing of blood vessels, which is particularly beneficial for surgeons in whole tissue transplant procedures. The lead candidate electropositive hydrogels, called APC1, was demonstrated in anastomosis mice models to be well tolerated, biocompatible, and non-toxic.