Longer-lived Mouse Models for Studying Gaucher Disease

The invention is a novel longer-lived mouse model for Gaucher disease. Gaucher disease is a genetic disorder that results from deficiencies in the enzyme glucocerebrosidase (GBA). The use of animal models to study how the disease progresses has been invaluable in research of this disorder. However, existing mouse models have been limited due to early mortality because the GBA enzyme plays an important role in lysosomal storage.

Multiplexing Homocysteine in Primary Newborn Screening Assays Using Maleimides as Select Derivatization Agents

Homocystinuria (HCU), a group of inherited disorders, causes symptoms ranging from failure to thrive and developmental delays in infants or young children to abnormal blood clots with onset in adults.1 Approximately 1 in 200,000 to 335,000 people have HCU globally.2

Minibody for Conditioning prior to Hematopoietic Stem Cell and Progenitor Cell Transplantation

Patient conditioning is a critical initial step in hematopoietic stem and progenitor cell (HSPC) transplantation procedures to enable marrow engraftment of infused cells. Conditioning regimens have traditionally been achieved by delivering cytotoxic doses of chemotherapeutic agents and radiation. However, these regimens are associated with significant morbidity and mortality, and cannot be used safely in elderly or subjects with comorbidities.

Trans-auricular Left Atrial Appendage Ligation to Prevent Thrombosis

This technology includes an interventional device to occlude the left atrial appendage to prevent thrombus formation. Atrial fibrillation is the most common cardiac arrhythmia and is associated with formation of thrombus in the left atrial appendage. Standard preventative treatment involves anticoagulation, which is not tolerated by all patients. Existing devices necessitate improvement because they need trans-septal puncture and anticoagulation to prevent thrombus or are prone to life-threatening complications.

Live Attenuated Vaccine to Prevent Disease Caused by West Nile Virus

West Nile virus (WNV) has recently emerged in the U.S. and is considered a significant emerging disease that has embedded itself over a considerable region of the U.S. WNV infections have been recorded in humans as well as in different animals. From 1999-2014, WNV killed 1,765 people in the U.S. and caused severe disease in more than 41,762 others. This project is part of NIAID's comprehensive emerging infectious disease program.

Monoclonal Antibodies That Bind to the Underside of Influenza Viral Neuraminidase

Current influenza vaccines mainly induce antibodies against the surface glycoprotein hemagglutinin (HA) that block viral attachment to its host receptors and viral membrane fusion to the host cell. The immunodominant head region of HA undergoes antigenic drift and antibodies directed to the head confer little cross-protections between strains or subtypes.

Protecting Healthcare Workers by Detecting Contamination From Hazardous Antineoplastic Drugs

CDC NIOSH early technology to detect surface contamination by hazardous antineoplastic drugs. Antineoplastic drugs, also known as anti-cancer drugs or chemotherapy, are used in the treatment of many types of cancer. While these drugs are lifesaving to patients, they must be handled with care by healthcare workers. Exposure from contaminated surfaces and drug vials can cause skin problems, birth defects, reproductive issues, and increased risk of various cancers.

Protecting Healthcare Workers by Detecting Contamination From Hazardous Antineoplastic Drugs

CDC NIOSH early technology to detect surface contamination by hazardous antineoplastic drugs. Antineoplastic drugs, also known as anti-cancer drugs or chemotherapy, are used in the treatment of many types of cancer. While these drugs are lifesaving to patients, they must be handled with care by healthcare workers. Exposure from contaminated surfaces and drug vials can cause skin problems, birth defects, reproductive issues, and increased risk of various cancers.