Immunogens for Use in a High Efficacy HIV Vaccine

Human immunodeficiency virus (HIV) infections remain a pandemic, most prevalent in Africa and the Americas. Anti-retroviral treatments have been effective in preventing spread of the virus and active outbreaks of acquired immune deficiency syndrome (AIDS). However, the development and deployment of an effective vaccine would provide long-lasting protection and alleviate the need to depend heavily on prevention methods that require continued access and adherence.

New Heterocyclic Scaffold-Based Inhibitors of the Polo-Box Domain of Polo-like Kinase 1 for the Treatment of Cancer

Polo-like kinase 1 (Plk1), a member of the Polo-like kinase family, plays a critical role in regulating mitosis and cell cycle progression. Aberrant expression of Plk1 has been observed in a variety of human cancers, and it is known to be associated with tumorigenesis as well as poor prognosis in cancer patients. Unlike normal cells, some cancer cells are dependent on augmented Plk1 levels to remain viable and are killed when Plk1 function is attenuated.

Improved Gene Therapy Vectors for the Treatment of Glycogen Storage Disease Type Ia (GSD-1a)

GSD-Ia is an inherited disorder of metabolism associated with life-threatening hypoglycemia, hepatic malignancy, and renal failure caused by the deficiency of glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC). Current therapy, which primarily consists of dietary modification, fails to prevent long-term complications in many patients, including growth failure, gout, pulmonary hypertension, renal dysfunction, osteoporosis, and hepatocellular adenomas (HCA).

High-Throughput Generation of Induced Pluripotent Stem Cells Carrying Antigen-Specific T Cell Receptors from Tumor Infiltrated Lymphocytes

One form of adoptive T cell therapy (ACT) consists of harvesting tumor infiltrating lymphocytes (TIL), screening and isolating TIL which display tumor antigen-specific T-cell receptors (TCR), expanding the isolated T cells in vitro, and reinfusing them into the patient for treatment. While highly active in the treatment of certain cancers (e.g., melanoma), current methods used to produce cancer-reactive T cells require significant time and may not adequately identify the desired TCRs which bind cancer targets.

Size-dependent brain distribution of macromolecular drug delivery platform

The blood brain barrier (BBB) is a specialized endothelium that prevents the uptake of substances from the systemic circulation into the central nervous system. This barrier, while protecting the sensitive physiological environment of the brain, is also a major impediment in administering therapeutics that need to pass through the BBB. A drug delivery platform that could deliver therapeutic agents directly to the brain is needed, and could have wide ranging significance in a variety of psychiatric, oncology, infectious, and neurodegenerative diseases.

Monomeric and Oligomeric Compounds as Contraceptives and Endocrine Therapeutics

The options for male contraceptives are limited. Research is ongoing to develop a male contraceptive based on hormonal activity. Testosterone is one of the hormones necessary in producing sperm.  Testosterone is absolutely required as a hormone for male fertility. Derivatives of testosterone for male contraceptives currently in clinical trials are associated with estrogenic deficiency. This deficiency can cause several issues including, but not limited to, bone density loss, risk of obesity, cardiovascular disease, and/or ineffective carbohydrate or lipid metabolism. 

RNASEH-Assisted Detection Assay for RNA

Several viral epidemics – such as the epidemics caused by H1N1 influenza virus, human immunodeficiency virus (HIV), Ebola virus, Zika virus, severe acute respiratory syndrome (SARS) virus, Middle East respiratory syndrome (MERS) virus and SARS-CoV-2 – have profoundly impacted global human health. Early identification of infected and/or infectious persons and isolating them from the population are some of the most effective and evident measures to prevent human-to-human spreading.

Composite Gels and Methods of their Use in Tissue Repair, Drug Delivery, and as Implants

Gel materials, particularly hydrogels, typically lose their mechanical strength and stiffness as they swell. This property  limits their use in both biological (e.g., cartilage and ECM repair) and non-biological (e.g., sealant) applications. Innovative materials in both medical and non-medical application areas are sorely needed.

Margaric Acid Decreases PIEZO2 Mediated Pain

Some existing therapies for treatment of pain are administered systematically and have significant side effects, such as addiction and drowsiness. Alternative therapy that does not impair normal touch function could be used to treat pain caused by mechanical injury or chronic inflammation. Administration of margaric acid was shown to ameliorate pain in mouse models of pain. In vitro data shows that margaric acid counteracts PIEZO2 (Piezo-type mechanosensitive ion channel component 2) potentiation evoked by bradykinin (i.e.

Extremely Rapid Method to Isolate Neoantigen Reactive T Cell Receptors (TCRs)

Adoptive cell transfer (ACT) uses tumor infiltrating lymphocytes (TILs) that recognize unique antigens expressed by cancer cells (“neoantigens”). Neoantigen specific TIL administration in patients has resulted in long term regression of certain metastatic cancers. However, one of the challenges of ACT and engineered T cell receptor (TCR) therapies more broadly, is the identification and isolation of these mutation specific TILs and TCRs. Only a fraction of TILs in a given patient is known to be tumor reactive, while the majority are not useful for cell therapy.