RNA interference (RNAi) is a naturally occurring cellular post-transcriptional gene regulation process that utilizes small double-stranded RNAs to trigger and guide gene silencing. By introducing synthetic RNA duplexes called small-interfering RNAs (siRNAs), we can harness the RNAi machinery for therapeutic gene control and the treatment of various diseases.
Antibody drug conjugates (ADC) can demonstrate high efficacy as cancer therapeutics, however, much more can be done to improve their efficacy and safety profile. Site-specific antibody drug conjugation is a promising way to do this. Scientists at the NCI’s Laboratory of Experimental Immunology have identified a fully human monoclonal antibody, m860, that binds to cell surface-associated Her2 with affinity comparable to that of Trastuzumab (Herceptin) but to a different epitope.
Adoptive immunotherapy is a promising new approach to cancer treatment that engineers an individual''s innate and adaptive immune system to fight against specific diseases, including cancer with fewer side-effects and more specific anti-tumor activity in individual patients. T cell receptors (TCRs) and Chimeric Antigen Receptors (CARs) are proteins that recognize antigens in the context of infected or transformed cells and activate T cells to mediate an immune response to destroy abnormal cells.
A child's growth is dependent on the proper functioning of the growth plate, a specialized cartilage structure located at the ends of long bones and within the vertebrae. The primary function of the growth plate is to generate new cartilage, which is then converted into bone tissue and results in the lengthening of bones. Failure of the growth plate to function properly can result in short stature or sometimes a skeletal dysplasia, such as achondroplasia, in which the bones are not just short but also malformed.
One major challenge in the development of effective cancer therapies is a lack of universal, cancer specific markers in target cells. The current standard therapies rely on surgery, chemotherapy, and radiation therapy. Such procedures lead to a population of resistant cancer cells that makes further applications of chemotherapy/radiation therapy ineffective. Additionally, the systemic application of chemotherapy lacks specificity and has off-target systemic effects that lead to adverse side effects.
Nitric oxide (NO) has a broad spectrum of actions in physiological and pathological processes. NO-donor drugs have shown therapeutic effect in several cancer types by inducing apoptosis but the concentrations required have suggested limited clinical applicability. For cancers such as non-small cell lung cancer where most therapies are not curative, there remains a need for effective treatments.
A number of factors can play a detrimental role in the process of wound healing such as poor nutritional status, smoking, various drugs, cancer, and diabetes. Wound healing impairment is a challenging clinical problem with no efficacious treatments currently available. Nitric oxide (NO) has been shown to play a role in the process of wound healing by promoting both the proliferative and remodeling phases of healing.
Thymoma and thymic carcinomas are a rare and poorly understood group of malignancies. Despite the growing number of biomarkers that are used for diagnosing and treating carcinomas in general, cancers of the thymus are still diagnosed, stratified and treated by a costly combination of histology, surgery and radiological procedures. The lack of qualified biomarkers associated with thymomas and thymic carcinomas has also hampered the development of targeted therapies.
Scientists at the National Cancer Institute's Molecular Targets Laboratory have discovered that Cnidarins as a novel class of highly potent proteins capable of blocking the HIV virus from penetrating T-cells. Cnidarins were found in a soft coral collected in waters off Australia's northern coast. Cnidarins can block virus fusion/entry but do not block viral attachment. In addition, Cnidarins do not have lectin-like activity and therefore possibly a unique mechanism of action.
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.