Multifunctional RNA Nanoparticles as Cancer and HIV Therapeutics

The promise of RNA interference based therapeutics is made evident by the recent surge of biotechnological drug companies that pursue such therapies and their progression into human clinical trials. The present invention discloses novel RNA  and RNA/DNA nanoparticles including  multiple siRNAs, RNA aptamers, fluorescent dyes, and proteins. These RNA nanoparticles are useful for various nanotechnological applications.

Nanoparticles for the targeted treatment of infected cells

Current treatments for cancer and viral infection are limited remedies that often suppress cell or viral replication rather than eliminate diseased cells entirely from the body. A further limitation is that these therapies often compromise healthy cells as well, leaving problems of recurrence and side effects.

Researchers at developed a novel therapeutic nanoparticle (NP) system harboring therapeutic small siRNA that can significantly enhance effectiveness and specificity of treatments by killing diseased cells.

Human T Cell Receptors for Treating Cancer

T cell receptors (TCRs) are proteins that recognize antigens in the context of infected or transformed cells and activate T cells to mediate an immune response and destroy abnormal cells. TCRs consist of two domains, one variable domain that recognizes the antigen and one constant region that helps the TCR anchor to the membrane and transmit recognition signals by interacting with other proteins. When a TCR is stimulated by an antigen, such as a tumor antigen, some signaling pathways activated in the cell lead to the production of cytokines, which mediate the immune response.

Methods for Selection of Cancer Patients and Predicting Efficacy of Combination Therapy

Available for licensing from the Laboratory of Cancer Biology and Genetics of the National Cancer Institute (NCI) is a novel gene signature of thirty-seven drug-responsive genes that links changes in gene expression to the clinically desirable outcome of improved overall survival. Expression of these genes has been linked to prognosis in several cancers, including, but not limited to: multiple myeloma, melanoma, and lung and breast cancers.

Human Antibodies Against Middle East Respiratory Syndrome Coronavirus

No effective therapeutics or vaccines against Middle East Respiratory Syndrome Coronavirus (MERS-CoV) are available. The human-to-human aspect of transmission and the high mortality rate associated with MERS-CoV infection have raised concerns over the potential for a future MERS-CoV pandemic and emphasized the need for development of effective therapeutics and vaccines.

Methods of Treating Giardiasis Using FDA-Approved Compounds

This technology includes a group of at least twenty-nine, diverse, commercially available compounds that are newly identified for activity against Giardia lamblia parasites. At least six of the candidate compounds, Bortezomib, Decitabine, Hydroxocobalamin, Amlexanox, Idarubicin, and Auranofin have preexisting FDA approval for human use for other (non-Giardia) conditions. Another three compounds, Fumagillin, Nitarsone and Carbadox have preexisting approval for veterinary use for non-Giardia conditions.

IL7Rα-Specific Antibody for Treating Acute Lymphoblastic Leukemia (ALL)

Acute lymphoblastic leukemia (ALL) is the most common cancer in children with approximately 3,250 new cases occurring per year in the United States. About 20% of cases are refractory to current treatment protocols and there is a desperate need for targeted therapies that do not result in adverse side effects such as cognitive impairment.