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.

Efficient mRNA-Based Genetic Engineering of Human NK Cells with High-Affinity CD16 and CCR7

A highly efficient method to genetically modify natural killer (NK) cells to induce expression of high affinity CD16 (HA-CD16) through mRNA electroporation, to potentiate NK cell-mediated antibody-dependent cellular cytotoxicity (ADCC). ADCC is mediated by CD16+ NK cells following adoptive NK cell transfer, but most humans express CD16 which has a relatively low affinity for IgG1 antibodies.

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.

Inner Curvature Charge Concentration Device for Tissue Laceration

Left ventricular outflow tract obstruction is a life-threatening complication of transcatheter mitral valve replacement caused by septal displacement of the anterior mitral leaflet (AML). The AML is a mobile structure that physically separates inflow and outflow zones of the left ventricle. Preserving the AML during surgical mitral valve replacement can cause left ventricular outflow tract obstruction, either when the prosthesis struts protrude into the left ventricular outflow tract or when along redundant anterior leaflet prolapses into the left ventrical outflow tract.

Potential Treatment for sickle-cell disease and thalassemia

The technology addresses treatment options for diseases such as sickle-cell and thalassemia. Traditionally, such beta-globinopathies are treated through bone marrow transplantation. However, this method is limited due to high treatment costs and finding a matched-donor. This relies on increasing fetal hemoglobin to potentially cure the disease. NIH inventors have identified a protein called Rio-Kinase 3 (RIOK3), that inhibits the production of fetal hemoglobin. Their work shows that inhibiting RIOK3 increases the production of fetal hemoglobin.

Local Magnetization Spoiling Using a Gradient Insert for Reducing the Field of View in Magnetic Resonance Imaging

The present invention provides a method and device for eliminating alias artifacts encountered in MRI when the field of view is made smaller than the subject being imaged. Significant advantages accrue from reducing the field of view to a smaller region of interest. These include reduced imaging time, increased spatial and temporal resolution, and less susceptibility to motion artifacts. The device operates by dephasing the magnetic resonance signal in regions away from the region of interest by means of a gradient insert.

Potentiating Antibody Therapy by Targeting Complement Deposited on Cancer Cells

Monoclonal antibodies (mAbs) have become a mainstay of therapy for many cancers. However, antibody therapy is not completely effective in some applications due to loss of the target surface antigen on cancer cells. Such mAb-induced “escape variants” are no longer sensitive to the therapeutic mAb therapy. It was observed that the escape variants carried covalently bound complement activation fragments, especially C3d. NIH inventors have generated several C3d-specific mouse and rabbit monoclonal antibodies to re-target cells that have escaped from mAb therapy.