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.

Agonist Epitopes for the Development of a Human Papillomavirus (HPV) Therapeutic Vaccine

Human papillomavirus (HPV) has been associated with the cause of several cancer types, including cervical, anal, and head and neck cancers. There has been great success in preventing HPV infections with the development of prophylactic HPV vaccines, Gardasil and Cervarix. However, these vaccines have only been shown to prevent HPV infection and not treat those already infected with HPV. These vaccines elicit antibody responses to late HPV genes, and thus would not be effective in treating established tumors.

Bioluminescent Bladder Cancer Cell Line for Tracking Cancer Progression

Bladder cancer is the fifth most common cancer in the United States and one of the costliest cancers to treat. Compared to other cancer types, bladder cancer has been understudied, and there is a need for informative mouse bladder cancer models that resemble the clinical situation and allow for evaluation of chemotherapeutic or immunotherapeutic agents. The orthotopic murine bladder cancer model MB49 resembles non-muscle invasive, nonmetastatic urothelial carcinomas and provides an opportunity to study the anti-tumor effects of immune cell checkpoint inhibitors.

Vascularized Thyroid-on-a-Chip for Personalized Drug Screening and Disease Modeling

This technology includes a micro-engineered “thyroid-on-a-chip” that combines human thyroid organoids with integrated micro-vasculature to replicate the gland’s native blood flow and 3-D architecture, enabling rapid, patient-specific drug screening. By permitting real-time perfusion of nutrients, hormones, and immune cells, the platform yields more physiologically relevant data than conventional static cultures or animal surrogates.

Evans Blue Modified Small Molecule-based Prostate-specific Membrane Antigen (PSMA) Radiotherapy and Nuclear Imaging

This technology includes anti-PSMA antibody labeled with 177Lu, which has shown to be an effective treatment for prostate cancer. Several small molecules targeting PSMA were also evaluated in prostate cancer patients labeled with betta emitters such as 177Lu. The most common one is 177Lu-PSMA-617 which is under clinical evaluation in many countries. Usual treatment in patients in most clinical trials was composed of up to 3 cycles of 177Lu-PSMA-617.

Innovative Antibody Conjugates for Targeted Therapy

This advanced technology introduces innovative antibody conjugates that redefine the possibilities of targeted therapy. By coupling therapeutic agents to engineered antibodies with highly specific binding sites, these conjugates deliver treatments directly to diseased cells while sparing healthy tissues. The result is a powerful increase in treatment efficacy, accompanied by a meaningful reduction in side effects.

Rabbit Antisera to Various Matrix, Matricellular, and Other Secreted Proteins

The extracellular matrix (ECM) is composed of a group of proteins that regulate many cellular functions, such as cell shape, adhesion, migration, proliferation, and differentiation. Deregulation of ECM protein production or function contributes to many pathological conditions, including asthma, chronic obstructive pulmonary disease, arthrosclerosis, and cancer. Scientists at the NIH have developed antisera against various ECM components such as proteoglycan, sialoprotein, collagen, etc.. These antisera can be used as research tools to study the biology of extracellular matrix molecules.