Stage of Program: Translational Tool/Bottleneck
Novel vectors for gene transfer into human ES cells
Human embryonic stem cells have a great potential for medical therapeutics. However, the genes required for altering the fate of these cells to differentiate into a particular tissue or cell type is not well understood. The ability to efficiently transfer genes or silence genes in ES cells would be of great benefit for two reasons: […]
A method to maintain and propagate pluripotent human ES cells
Human embryonic stem (hES) cells are pluripotent such that they can differentiate into all three germ layers, thus potentially all different types of tissues of the body. Pluripotency is characteristic of only embryonic cells, but it can also be achieved by reprogramming differentiated cells by transferring nuclear contents into unfertilized, enucleated oocytes or by fusing […]
Technology for hESC-Derived Cardiomyocyte Differentiation and Optimization of Graft-Host Integration in Adult Myocardium
Stem cells therapies hold great promise in the treatment of cardiac diseases such as coronary heart disease or congestive heart failure. Thanks to their ability to transform into almost any kind of tissue, engrafted stem cells can potentially replace damaged heart tissues with healthy tissues, effectively restoring the heart’s original functions. While initial studies demonstrated […]
Identifying roadblocks to neural stem cell transplantation into human tissues.
Research Objective We will generate a comprehensive map of human neural stem cell differentiation profiles that will serve as a reference for enhancing neural stem cell-based therapies. Impact Our project will develop improved protocols for human neural stem cells differentiation, enhancing the fidelity, safety and robustness of future cell therapies. Major Proposed Activities Establish quantitative […]
Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies
Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]
Autologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cells
Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury
Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human disease. Impact OPCs have therapeutic potential for spinal cord injury, restoration of cognitive function after cancer radiation therapy, inherited demyelinating disease, and potentially multiple sclerosis. […]
Purification of Human Hematopoietic Stem Cells (HSCs) for Clinical Stem Cell Transplantation
Translational Candidate Two new monoclonal antibodies anti-CD34 and anti-CD90, and protocols to purify cancer-free and/or T cell free human HSC for clinical transplantation. Area of Impact The development of hematopoietic stem cell-based therapies, starting with rescue of metastatic breast cancer patients with their own cancer-free HSC Mechanism of Action The mechanism is using highly purified […]
Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing
Translational Candidate HiDef-B8, an iPS cell maintenance medium designed to reduce stem cell GMP manufacturing costs and risk. Area of Impact HiDef-B8 addresses scale-up manufacturing, by being lower-cost ($300/L) and requiring fewer passages per week (1-2 as opposed to 3-5). Mechanism of Action HiDef-B8 underwent extensive empirical optimization and alternative component screening, focused on reducing […]
Generation of human universal donor iPS cells
Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection by host immune cells. Mechanism of Action The universal donor cell mitigates immune rejection by host immune cells and is equipped with a suicide gene […]