Systemic CRISPR Therapy for the Treatment of Inherited Diseases

This technology includes novel systemic adeno-associated virus (AAV)-mediated CRISPR gene therapy technology. While some diseases (e.g., retinal diseases) can be treated through local gene transfer, many diseases such as Duchenne Muscular Dystrophy (DMD) require systemic therapy. The CRISPR technology has two components, the Cas9 endonuclease, and the gRNA. To explore systemic CRISPR therapy, we co-delivered the AAV.Cas9 and AAV.gRNA vector to mdx mice, a mouse DMD model. Direct delivery to muscle yielded efficient gene correction.

T-cell Receptor Targeting Human Papillomavirus-16 E7 Oncoprotein

Human papillomavirus (HPV) is a group of human viruses known to cause various malignancies. Of the group, HPV-16 is the most prevalent strain – an estimated 90% of adults have been exposed. HPV-16 is also the strain most commonly associated with malignancy, causing the vast majority of cervical, anal, vaginal, vulvar, and penile cancers. Currently, HPV-positive malignancies non-responsive to surgery or radiation are incurable and poorly palliated by existing systemic therapies. Thus, an alternative therapeutic approach for HPV-positive malignancies is needed. 

T-cell Receptor Targeting Human Papillomavirus-16 E6 Oncoprotein

Human papillomavirus (HPV) is a group of human viruses known to cause various malignancies. Of the group, HPV-16 is the most prevalent strain – an estimated 90% of adults have been exposed. HPV-16 is also the strain most commonly associated with malignancy, causing the vast majority of cervical, anal, vaginal, vulvar, and penile cancers. Currently, HPV-positive malignancies non-responsive to surgery or radiation are incurable and poorly palliated by existing systemic therapies. Thus, an alternative therapeutic approach for HPV-positive malignancies is needed. 

 

Use of Antihistamine Compounds for the Treatment of Hepatitis C Virus

The vast majority of people infected with Hepatitis C Virus (HCV) will have chronic infection. Over decades, this can lead to liver disease and liver cancer. In fact, HCV infection is the leading cause of liver transplants in the U.S. Several new drugs have recently come into the market that will likely change the HCV treatment paradigm. However, the effectiveness of these new drugs can vary depending on the HCV genotype. Thus, there is still the need for additional new therapeutics against HCV.

In-vivo System to Interrogate the Functions of Mucous Membranes and Identify Mucin/Glycan Mimetics and JAK/STAT Inhibitors for the Treatment of Diseases of the Oral Cavity and Digestive Tract

This technology includes a Drosophila mutant strain that can be used as an in vivo model for diseases of the oral cavity and digestive tract (Sjogren's syndrome, colitis, colon cancer, inflammatory bowel disease), where the mucous membrane is disrupted or non-functional. This mutant lacks a mucous membrane and displays epithelial cell damage, uncontrolled cell proliferation and the up-regulation of conserved signaling pathways (JAK/STAT).

Antigen Mixtures for Serological Detection of HHV-8 Infection

This invention describes a highly specific and sensitive serological test for human herpesvirus 8 (HHV-8) infection that uses the Luciferase Immunoprecipitation System (LIPS). A mixture of four virus-specific antigens, including K8.1, v-cyclin, ORF65 and LANA, was shown to provide more robust detection of HHV-8 infection than traditional methods due its ability to detect very low viral loads.

Simple, Quantitative Sensitive High-throughput Antibody Detection for Lyme Disease

This technology is for compositions and methods for diagnosis of Lyme disease. Currently, Lyme disease is diagnosed by clinical exam and a history of exposure to endemic regions. Although, laboratory tests may aid diagnosis, the best tests currently available are slow and labor intensive and require understanding of the test, and infection stage. A two-step antibody based test process is currently the recommended laboratory test. The first step is either an enzyme immunoassay (EIA), or an indirect immunofluorescence assay (IFA).

Novel Antigen for Use as Vaccine Against Nematode Infection

This invention describes a new vaccine against Strongyoides stercoralis, which establishes a parasitic infection that affects an estimated 100-200 million people worldwide. The potential for fatal disease associated with S. stercoralis infection and the difficulty in treating hyperinfection underscores the need for prophylactic vaccines against the disease. This vaccine uses S. stercoralis immunoreactive antigen (SsIR); a novel antigen capable of providing 70-90 % protection for mice immunized with the antigen.

Recombinant NIE Antigen from Strongyloides stercoralis

Strongyloides stercoralis is an intestinal nematode endemic that affects an estimated 30 to 100 million people worldwide. Many of these individuals may be asymptomatic for decades. The present invention discloses a NIE recombinant antigen that can be used in improved assays and diagnostics for S. stercoralis infection. The NIE antigen is the only one that is non-cross-reactive with sera from humans with other related filaria infections. The NIE antigen can be utilized as a skin test antigen for immediate hypersensitivity as well as for use in ELISA or other assays.
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