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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).

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.

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. 

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.

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.

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.

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.

Adoptive cell transfer (ACT) uses tumor infiltrating lymphocytes (TILs) that recognize antigens expressed by cancer cells (neoantigens). Neoantigen specific TIL administration in patients has resulted in long-term regression of certain metastatic cancers. However, current procedures for TIL therapy are highly invasive, labor-intensive, and time consuming. The success of these procedures is limited and differs between patients and histologies.

Pancreatic ductal adenocarcinoma (PDA) accounts for more than 90% of pancreatic cancer cases, and it is one of the most aggressive malignancies with a 5-year survival rate of 6%. The high mortality rate caused by PDA is primarily from the lack of early diagnosis – it is often asymptomatic in early stages – and a poor response to conventional chemotherapy and radiotherapy. One of the major immune cell types present in the PDA microenvironment is a subset of macrophages commonly termed tumor-associated macrophages (TAM).

Prostate cancer is the most prevalent form of cancer among all men in the United States (US). It is also the second leading cause of cancer-related deaths in the US among men, largely due to the progressively treatment resistant nature of the disease. Treatment options for early stage prostate cancer include watchful waiting, radical prostatectomy, radiation therapy, and importantly androgen-deprivation therapy (ADT). Prostate cancer is dependent on androgen hormones, such as testosterone, for sustaining and promoting growth.