Summary:
The National Cancer Institute (NCI) seeks research co-development partners and/or licensees for engineered chimeric snoRNA guides that recruit NAT10 to a specific target and cause directed acetylation of the target. They could be used to treat haploinsufficiency-associated disorders or diseases.
Description of Technology:
The development of an effective HIV vaccine has been ongoing. HIV sequence diversity and immunodominance are major obstacles in the design of an effective vaccine. Researchers at the National Cancer Institute (NCI) developed a novel vaccine strategy combining both DNA and mRNA vaccination to induce an effective immune response. This combination strategy could also be used to develop vaccines against cancer or other infectious diseases (ex. SARS-CoV-2).
Tailoring the best treatments to cancer patients remains a highly important endeavor in the oncology field. However, personalized treatment courses are challenging to determine, and technologies or methods that can successfully be employed for precision oncology are lacking.
Nuclear medicine is the second largest source of medical radiation exposure to the general population after computed tomography imaging. Imaging modalities utilizing nuclear medicine produce a more detailed view of internal structure and function and are most commonly used to diagnose diseases such as heart disease, Alzheimer’s and brain disorders. They are used to visualize tumors, abscesses due to infection or abnormalities in abdominal organs.
About half of the per capita dose of radiation due to medical exposures is provided by computed tomography (CT) examinations. Approximately 80 million CTs are performed annually in the United States. CT scans most commonly look for internal bleeding or clots, abscesses due to infection, tumors and internal structures. Although CT provides great patient benefit, concerns exist about potential associated risks from radiation doses – especially in pediatric patients more sensitive to radiation.
Cyclin-dependent kinase inhibitor 2A gene, also known as CDKN2A, is a tumor suppressor gene and is commonly inactivated through somatic mutations in many human cancers. For example, inactivation of CDKN2A is highly prevalent in melanoma, gastrointestinal and pancreatic cancers. Through germline mutations, CDKN2A is associated with predisposition for a variety of cancers, including melanoma and pancreatic cancers. Despite the high frequency of CDKN2A mutations in cancer, there have been no successful therapies targeting these mutations to date.
Topoisomerase 1 (TOP1) is an essential enzyme that plays a critical role in DNA transcription and replication. TOP1 inhibitors are a known class of anti-cancer agents that work to interrupt DNA replication in cancer cells, causing cell death. Since the discovery of the TOP1 inhibitor camptothecin (CPT) from plant extracts more than 60 years ago, two CPT analogs (irinotecan and topotecan) were approved by the FDA for cancer treatment. Tyrosyl-DNA phosphodiesterase 1 (TDP1) is an enzyme involved in DNA repair created when TOP1 is inhibited.
Adoptive cell therapy (ACT) is a breakthrough form of cancer immunotherapy that utilizes tumor infiltrating lymphocytes (TILs) or genetically engineered T cells to attack tumor cells through recognition of tumor-specific antigens. A major hurdle in the development of ACT is the identification and isolation of T cells that recognize antigens that are expressed by tumor cells but not by healthy tissues. Current methods to identify such T cells involve extracting autologous antigen presenting cells (APCs) from patients in an expensive, laborious, and time-consuming process.
BRAF is an oncogene that encodinges a serine-threonine kinase (B-Raf kinase) important in regulating cell growth and differentiation. Spontaneous mutations in the BRAF gene allow cells to continuously divide, leading to the development of cancer. A substitution of glutamic acid for valine at amino acid number 600 (designated V600E) accounts for 90% of BRAF mutations and is a driver of many cancers. The V600E mutation is present in ~3% of all cancer cases, representing a patient population of 540,000 patients per year.
