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The Transforming Growth Factor Beta (TGF-ß) ligands (i.e., TGF-ß1, -ß2, -ß3) are key regulatory proteins in animal physiology. Disruption of normal TGF-ß signaling is associated with many diseases from cancer to fibrosis. In mice and humans, TGF-ß activates TGF-ß receptors (e.g., TGFBR1), which activates SMAD proteins that alter gene expression and contribute to tumorigenesis.  Reliable animal models are essential for the study of TGF-ß signaling.

Researchers at the National Cancer Institute’s Biopharmaceutical Development Program recently developed massively parallel sequencing methods for virus-derived therapeutics such as viral vaccines and oncolytic immunotherapies.

Lung metastases are a sign of widespread cancer with poor survival rate. Lung malignancies can originate from almost any cancer type spread via the blood stream. Most common lung metastases are from melanoma, breast cancer, bladder cancer, colon cancer, prostate cancer, neuroblastoma, and sarcoma. Living more than 5 years with lung metastases is uncommon, and surgical procedures are only effective with localized lung metastases. Lung metastasis are extremely frequent and resistant to regular treatment due to immunosuppressive regulatory sulfatide-reactive type II NKT cells.

Human immunodeficiency virus (HIV) remains a major global health challenge despite the advancement made in development of effective antiretrovirals (ARVs). ARVs are effective at limiting replication and spread of the virus, and progression to acquired immuno-deficiency syndrome (AIDS). However, ARVs often lead to emergence of drug-resistant virus strains insensitive to treatment and with toxic effects following long-term usage.

This technology includes a number of dimeric RGD peptides which been developed and labeled with various PET isotopes (1BF, 68Ga, and 64Cu) for imaging integrin expression in cancer, inflammation, rheumatoid arthritis, myocardial infarct, stroke and traumatic injury. A number of these peptides have been translated into clinic for diagnosis and therapy response monitoring.

This technology includes a small molecule drug (VDAC inhibitor, also known as VBIT4) that may be useful for inhibiting lupus disease. To test lupus animal model, VBIT4 was continuously administered for 5 weeks to mice and there was no mortality or clinical symptoms in these animals. Additionally, VBIT4 treatment blocked the development of skin lesions and alopecia of the ears and face, and suppressed the thickening of the epidermis that accompanies leukocyte infiltration.

This technology includes antibodies against TL 1A for the inhibition of TL 1A-DR3 interactions for the diagnosis and treatment of various autoimmune diseases. Through the use of our developed hamster anti-mouse and mouse anti-human monoclonal antibodies, we’ve demonstrated that treatment with anti-mouse TL 1A prevented collagen-induced arthritis and TNBS-induced colitis NEED TO UPDATE

This technology includes the use of genetic markers to predict the response of patients, particularly children with systemic juvenile idiopathic arthritis (sJiA), to anakinra treatment. Anakinra is a human recombinant IL-1 RA used in treating sJiA, a severe childhood inflammatory disease where early and effective treatment is essential for better long-term outcomes. Through the analysis of 38 children with sJiA treated with anakinra, specific sJiA-associated SNPs (single nucleotide polymorphisms) were identified as predictors of therapeutic failure, with a significant odds ratio of 17.3.

This technology includes the creation and use of A3 adenosine receptor (A3AR)-selective agonists for treating chemotherapy-induced peripheral neuropathy, chronic neuropathic pain, rheumatoid arthritis, psoriasis, and other conditions. A3 receptors for adenosine are found in most cells and endogenous activation of the A3 receptors can result in apoptosis, thereby relieving the inflammation or targeting a tumor. A3AR agonists have been a promising strategy for the treatment of various diseases.