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.

A Novel Therapeutic Vector for Hemoglobin Disorders

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector.

Emergency Maritime Battery Charger

Boats and other watercrafts have emergency lifesaving equipment like strobe lamps to help rescuers locate individuals overboard in the event of a disaster. The battery life of the equipment is limited, so the amount of time rescuers have to find the victims is also limited. An emergency battery charger that can power emergency equipment is needed to remove this limitation.

Swing-Away Winch Cathead Guard

Shrimp boat operators use two trawl retrieval mechanisms mounted on the same winch frame. The main spools are used in the first operation; the shrimpers stand within inches of rotating cathead drums and guide incoming wire rope onto the main spools. Second, lazy lines are wrapped multiple times around each of the two spinning catheads (horizontal beams that raise and secure the anchor). Lastly, the guarding ends are pulled by the operators to cinch the rope to the rotating spool aiding trawl retrieval, but poses a hazard for operators to get entangled in the spinning cathead spools.

Modified AAV5 Vectors for Enhanced Transduction and Reduced Antibody Neutralization

Scientists at the NIH disclosed a mutated adeno-associated virus (AAV) serotype 5 by modifying sialic acid binding regions which mediate viral entry into host cells. Preliminary results from animal studies suggest that this modification can increase transduction by 3-4 folds in salivary glands and muscles, and can significantly decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Thus, the modified AAV5 vectors seem to be optimal for gene therapy.

A Novel Adeno-Associated Virus for Gene Therapy

Scientists at the NIH disclosed a novel adeno-associated virus (AAV) termed "44-9." AAV44-9 based vectors have high gene transfer activity in a number of cell types, including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum). These vectors can increase the transduction efficiency and decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV.

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.

A Novel Rapid Point-of Care Diagnostic Method for Infectious and Autoimmune Diseases

Rapid point-of-care, antibody-based testing is not available for the diagnosis of autoimmune and most infectious diseases. For detecting autoantibodies associated with most autoimmune conditions, fluid-phase immunoprecipitation assays are required. However, these assays usually involve radioactivity and are not feasible for point-of-care applications. The subject invention describes methods of using neodymium magnet for diagnosis of infectious and autoimmune diseases including lupus, Sjögren's syndrome, type I diabetes, HIV and Lyme disease.

SIRT1 KO Human Cell Lines Generated by CRISPR/Cas9-mediated DNA Editing

SIRT1, a NAD+-dependent protein deacetylase, is the most conserved member of the sirtuins family. Through deacetylation of a number of protein substrates that are important transcription factors or co-factors, SIRT1 regulates many vital biological processes such as metabolism, cellular stress response, stem cell pluripotency, and development.
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