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.

Device for Closure of Transvascular or Transcameral Access Ports

This technology includes a novel method 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 nitinol occluder devices to occlude intracardiac holes and to allow non-surgical direct access to the heart. This new disclosure adds additional design features that have been tested in vivo.

Novel Bicuspid Transcatheter Heart Valve Frame and Leaflets for Mitro Valve Implantation

This technology includes a pair of subsystems for a novel transcatheter bicuspid valve (frame and leaflets) intended for implantation in the mitral position. It is simple, it overcomes key limitations to transcatheter bicuspid mitral valve implants, and it overcomes key limitations to transcatheter tricuspid mitral valve implants.

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.

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.

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).

CXCR4 Reduction Leads to Enhancement of Engraftment of Hematopoietic Stem Cells

Methods of enhancing engraftment of donor hematopoietic stem cells (HSCs) by reducing expression or activity of CXCR4 in HSCs is described. HSC are the only cells in the bone marrow that are both pluripotent and long lived. Bone marrow transplantation (BMT) using HSC is an increasingly common medical therapy for severe hematologic cancers and primary hematologic immunodeficiencies. However, for significant HSC engraftment to occur there must usually be pre-transplant conditioning with either irradiation or chemotherapy or both.
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