This technology includes rhesus macaque induced pluripotent stem cells (iPSCs) lines from multiple animals and various types of cells to establish this pre-clinical model. iPSCs are a type of pluripotent stem cell that can be generated from adult somatic cells. The iPSC technology holds great potential for regenerative medicine. Before clinical application, it is critical to evaluate safety and efficacy in a clinically-relevant animal model. We propose that non-human primate models are particularly relevant to test iPSC-based cell therapies.

This technology includes a novel transcatheter repair for functional mitral valve regurgitation, called mitral cerclage annuloplasty. This includes coronary artery protection for mitral cerclage annuloplasty against inside-out compression from subsequent transcatheter valve-in-ring mitral valve implantation, wherein the ring is created by the cerclage annuloplasty. Cerclage annuloplasty is to create a semi-rigid ring at the level of the mitral annulus.

This technology includes a metallic guidewire that is suitable for MRI catheterization, because it is mechanically long but electrically consists of short conductive segments that cannot resonate during MRI. The invention consists of stiffness-matched non-conductive connectors or connections that are used along with short metallic segments. The embodiment reduced to practice has torquability and flexibility comparable to marketed metallic guidewires, yet is free from MRI heating.

This technology includes a new reagent, termed bivalent Tn5 complex, and applied it to mapping genome-wide enhancer-promoter interactions to be utilized for disease diagnostics. Chromatin structure is critical for regulating transcription in normal development and disease states. In particular, the interaction between enhancers and promotes are essential for the temporospatial control of gene expression.

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.

This technology includes a catheter design to be used for cardiovascular procedures. This catheter has intrinsic freedom from MRI heating by segmenting and insulating the braiding material, yet that retains most of the mechanical properties of a braided catheter aligning the segment interruptions out-of-phase with each other.

This technology includes a catheter-delivered endograft designed to treat congenital heart disease without surgery. The specific surgical procedure averted is cavopulmonary bypass graft. The key innovations are features to effect distal end-to-side anastomosis and proximal end-to-end anastomosis without surgery. The system operates under X-ray and MRI guidance.

This technology includes monoclonal antibodies (mAb) that specifically and with high affinity bind the final complement components C3dg and C3d (subsequently referred to as C3d), which can be used to kill tumor cells that carry C3d on their cell surface. We show that tumor cells of patients treated with the therapeutic anti-CD20 mAb ofatumumab carry C3d on the cell surface and can bind and be killed by addition of anti-C3 mAbs. In contrast, further addition of more ofatumumab has only minimal effects.

This technology includes the use of engineered human induced pluripotent stem cells (iPSCs) for various applications such as studying cell differentiation, drug screening, and gene transfer therapy. It employs gene targeting donors flanked by DNA sequences compatible with endogenous loci to integrate transgenes through homologous recombination. A key aspect is the flexible gene targeting donor design, used in conjunction with safe harbor transcription activator-like effector nucleases (TALENs).

This technology includes two safe harbor gene targeting donors, specifically designed for applications in the study of induced pluripotent stem cells (iPSC). These include the pAAVS1D-CMV.RFP-EF1a.copGFPpuro and pAAVS1-iCLHN donors. A key feature of these donors is their ability to integrate various transgenes into specific loci through homologous recombination, facilitated by sequences homologous to safe harbor loci. When paired with TALENs targeting these loci, these plasmids enable precise and efficient genome engineering in human cells.