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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.

Software is has been developed and available for licensing that fully automates image processing for the quantification of myocardial blood flow (MBF) pixel maps from firstpass contrast-enhanced cardiac magnetic resonance (CMR) perfusion images. The system removes the need for laborious manual quantitative CMR perfusion pixel map processing and can process prospective and retrospective studies acquired from various imaging protocols. In full automation, arterial input function (AIF) images are processed for motion correction and myocardial perfusion images are corrected for intensity bias.

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.

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.

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.

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.

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.

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.

This technology includes the use of auranofin for the treatment of Chronic Lymphocytic Leukemia (CLL). Auranofin is currently approved for the treatment of rheumatoid arthritis and has been shown to display anti-cancer activity. CLL is a blood and bone marrow disease that usually progresses over a lengthy period of time and normally occurs in middle-age adults. The current therapeutic options for CLL patients are limited, and there are few therapies under development.

This technology includes the generation and use of human induced pluripotent stem cell (iPSC) lines that can be used to study and screen potential therapeutics for lysosomal storage diseases (LSDs). LSDs are a group of 50 genetic disorders caused by mutations in the genes encoding lysosomal enzymes and proteins. Although various therapeutic approaches exist, most cases of LSDs are not effectively treated due to a lack of therapeutics (including stem cells and recombinant proteins).