A Neural Stem Line from a Niemann Pick C (NPC) Type 1 Patient for Therapy Development

This technology includes a neural stem cell (NSC) line derived from a Niemann Pick C (NPC) patient, aimed at advancing research and drug development for NPC, an inherited neurodegenerative disorder characterized by the accumulation of cholesterol in neurons. The NSCs, which serve as a crucial intermediate cell type, can be differentiated into any neuronal or glial cell of the brain or spinal cord under appropriate culture conditions. These cells originate from fibroblasts reprogrammed into induced pluripotent stem cells.

Neural Stem Cells from an iPSC Line Ubiquitously Expressing Green Fluorescent Protein for Basic Science Research and Cell Line Tracking

This technology involves neural stem cells (NSCs) derived from pluripotent stem cells (PSCs) that can differentiate into neurons and glia. The key feature of this technology is the CY2 EEF1A1 GFP iPSC line, which includes a green fluorescent protein (GFP) expressed under the EEF1A1 promoter, leading to its ubiquitous expression in cells. This characteristic makes the NSCs and the neural cells differentiated from this line exhibit green fluorescence. Such cells, when transplanted into animal models like mice and rats, can be easily tracked due to their fluorescence.

Enhanced S10-3 Cell Line for Advanced Hepatitis E Virus Research and Therapeutic Development

The Huh-7 cell line underwent a detailed sub-cloning process to enhance its effectiveness for Hepatitis E Virus (HEV) infection studies. This involved diluting and culturing cells in 96-well plates until confluent monolayers formed, followed by selection and expansion of the most suitable cells. The sub-clone S10-3, derived from this process, was identified as the most efficient for transfection and infection by HEV.

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.

Device for Closure of Transvascular or Transcameral Access Ports

This technology includes part of transcatheter aortic valve replacement and to enable non-surgical thoracic aortic aneurysm endograft repair. The invention enables a completely new way to access the arterial circulation to allow introduction of large devices, such as transcatheter aortic valve replacement, percutaneous left ventricular assist devices, and thoracic aortic endografts. It also can be used in most labeled and off-label applications of Amplatzer (AGA Medical, St Jude) nitinol occluder devices to occlude intracardiac holes and to allow non-surgical direct access to the heart.

Endo-cameral Closure Device for Structural Heart Defects and Blood Vessel Repair

This technology includes a device to close a hole in the wall of a large blood vessel or cardiac chamber from the inside out, delivered over a guidewire and through a catheter or sheath. First, the proximal portion deploys within the vessel or chamber and is advanced over a guidewire to oppose the wall and seal the hole. Second, the distal portion self-assembles outside the vessel or chamber upon withdrawal of the guidewire. Deployment of the distal portion anchors the device securely in place.

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.

Development and Characterization of the SLC46A3 Knockout Mouse Line

Nonalcoholic fatty liver disease is caused by several factors including 2,3,7,8-tetrachlorodibenzo-p-dioxin (TCDD), an environmental contaminant. TCDD causes lipid accumulation in humans by inducing the Solute Carrier Family 46 Member 3 (SLC46A3) gene expression. To effectively study TCDD-mediated lipid accumulation, research tools such as SLC46A3 knockout cells and animal models are required.