Directed Acetylation of Cytidine in Cellular mRNA through Engineered snoRNA Adapters for the Treatment of Haploinsufficiencies

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: 

Combined RNA and DNA Vaccination Strategy for Improving the Vaccine Immune Response

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). 

Camel VHH Nanobodies Bind the S2 Subunit of SARS-CoV-2 and Broadly Neutralize Variants including Omicron

Since its emergence in 2019, COVID-19 infected over 600 million people and over 6 million people have died from the disease. COVID-19 is an infectious disease caused by the SARS-CoV-2 virus. Neutralizing antibodies have been developed to bind to the receptor binding domain (RBD) on the spike (S) protein. Blocking the interaction of the RBD and the ACE2 receptor, is critical in neutralizing the virus. However, the S2 subunit, is also critical for viral infection and entry into human cells.

Methods of Predicting Patient Treatment Response and Resistance via Single-Cell Transcriptomics of Their Tumors

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.

Novel Human Islet Amyloid Polypeptides as Alzheimer’s Disease Biomarkers and Inhibitors of Amyloid Formation

Over 34 million Americans are living with diabetes. An estimated 6.5 million Americans are living with Alzheimer’s disease (AD) and type 2 diabetes mellites (T2DM). Amyloidosis due to aggregation of amyloid-β is key pathogenic event in AD, whereas aggregation of mature islet amyloid polypeptide (IAPP37) in human islet leads to β-cell dysfunction. A hallmark feature of T2DM is the accumulation of islet amyloid polypeptide fibrils in pancreatic islets. Such accumulations form amyloid plaques and cause apoptosis of -cells of islets. 

T Cell Receptor Targeting CD22 for the Treatment of Lymphomas and Leukemias

CD22 is a protein expressed by normal B cells and B-lymphoid malignancies. Its limited tissue expression pattern makes it a safe antigen for targeted therapies, such as T-cell Receptor (TCR)-T cell therapy. CD22-targeting therapies already on the market, mainly antibody-immunotoxin conjugates and chimeric antigen receptors (CAR)-T cells, have limitations such as resistance to treatment and/or side effects. Resistance mechanisms to the current CD22 therapies involve loss or modulation of target antigen on the cell surface.

High-Throughput Assay for Detection and Monitoring of Endocrine Disrupting Chemicals in Water Sources

There is growing awareness that a wide variety of synthetic and natural compounds that may be present in water sources, such as streams, wells, and ground water, may lead to adverse health effects, including increased cancer risk. Even low concentrations of these compounds are of concern, as they may have biological effects at concentrations of parts per billion or less.

A Human Monoclonal Antibody Against Deacetylated PNAG for Use as an Antimicrobial Agent

Biofilms are complex microbial communities, surface attached and held together by self-produced polymer matrices.  These matrices are mainly composed of polysaccharides, secreted proteins and nucleic acids.  Poly-N-acetyl glucosamine (PNAG) is a highly conserved surface polysaccharide expressed by a range of bacterial, fungal and protozoan microorganisms.

FIBP Knockout Potentiates Therapeutic Effects of T-cell Based Therapies in Solid Tumors

Despite recent breakthroughs in cancer immunotherapy, T-cell based therapies achieve limited efficacy in solid tumors. Immunosuppression, antigen escape and physical barriers to entry into solid tumors are issues faced. Identifying regulators in T-cell dysfunction remains challenging due to limitations of current screening platforms. 

Method for Assembling Decellularized Tissue Extracellular Matrix in 3D Tumor Spheroids

Cell culture investigations using spheroids and organoid models have had a major impact on biomedical advancement as alternative sources for costly, in vivo animal testing.  However, these 3-D cell constructs are limited in that they do not integrate extracellular components within the structure important for more reliable and accurate biological responses.  Extracellular matrix (ECM) from decellularized tissues provide a physical scaffolding and offers crucial biochemical and biomechanical cues for cellular constituents.