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.

Genome Wide DNase I Hypersensitive Sites Detection in Formalin-Fixed Paraffin-Embedded Single Cells

A method of detecting DNase I hypersensitive sites ((DHS) in a single cell or very small number of cells, including cells recovered from formalin-fixed paraffin-embedded (FFPE) tissue slides of patient samples. DHS has revealed a large number of potential regulatory elements for transcriptional regulation in various cell types. The application of DNase-Seq techniques to patient samples can elucidate pathophysiological mechanisms of gene function in a variety of diseases as well as provide potentially important diagnostic and prognostic information.

Three-Dimensional Curved Catheter for Right Atrial Appendage Traversal

Available for licensing and commercial development is a three-dimensionally configured curved catheter for safe traversal of the right atrial appendage (RAA). The device is configured to optimize one-way access of the pericardial space through the right atrium and into the RAA reducing the risk of coronary lacerations. Specifically the curved catheter is best described in three segments: a proximal segment, a transitional segment and a distal segment; the transition segment having a clockwise spiral shaped curvature.

Separation Of Polar Compounds By Affinity Countercurrent Chromatography

A new and highly advantageous method of purifying polar organic compounds using affinity countercurrent chromatography, has been created. This invention permits separation of very hydrophilic organic compounds using countercurrent chromatography in which a ligand for the desired analytes is used to enhance the partitioning of polar species into the organic layer of an aqueous-organic solvent mixture.

Alloreactive T Cell Depletion Method For Preventing Graft-Versus-Host Disease

The invention relates to the use of adenosine to deplete alloreactive T cells from donor grafts to prevent graft-versus-host disease (GVHD). The method includes culturing donor cells that include T cells with recipient antigen presenting cells (APCs) to form a mixture of cells. The recipient’s APCs activate donor T cells. The activated T cells are treated with high doses of adenosine or an adenosine-like molecule to decrease or inhibit viability of the activated donor T-cells.

Capsid-Free AAV Vectors for Gene Delivery and Their Use for Gene Therapy

The invention concerns novel capsid-free AAV vectors that can be used for gene delivery and gene therapy applications. The invention provides for a linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest, and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response.

Enhanced Functionalization of Carbon Nanoparticles for Biomedical Applications

The invention pertains to methods of increasing the density of carboxylic acids on the surface of a carbon nanoparticle that can be functionalized with biologically relevant molecules, such as antibodies or peptides, for biomedical applications. Advantageously, the method could increase functionalization of a nanoparticle by at least about 1x107 functional groups/g of nanoparticle.

Human and Veterinary Cancer Therapeutic Agent Utilizing Anthrax Toxin-Based Technology

Due to the disorganized nature of blood vessels that run through tumors, chemotherapeutic agents often fail to penetrate tumors and kill cancer cells at the tumor’s center. This can lead to ineffective chemotherapeutic treatments, because tumors can quickly grow back if the entire tumor is not destroyed. NIH researchers have developed a therapeutic agent that solves this problem facing current chemotherapy treatments.

Hybrid Computer Tomography Scanning System

The invention relates to a combination hybrid computer tomography (CT) system that is particularly suited for elucidating stages in pulmonary diseases, notably cystic fibrosis and lung cancer. Improved visualization of lung parenchyma and the margins of lung cysts (non-invasive “virtual biopsy”) may provide sufficient detail to distinguish the types of cystic lesions such that the typical lung tissue pathologic biopsy would not be needed to make a diagnosis.

Human and Veterinary Cancer Therapeutic Agent Utilizing Anthrax Toxin-Based Technology

Due to the disorganized nature of blood vessels that run through tumors, chemotherapeutic agents often fail to penetrate tumors and kill cancer cells at the tumor’s center. This can lead to ineffective chemotherapeutic treatments, because tumors can quickly grow back if the entire tumor is not destroyed. NIH researchers have developed a therapeutic agent that solves this problem facing current chemotherapy treatments.