Stage of Program: Basic Research and Discovery Stage Programs
Engineered Biomaterials for Scalable Manufacturing and High Viability Implantation of hPSC-Derived Cells to Treat Neurodegenerative Disease
Cell replacement therapies (CRTs) have considerable promise for addressing unmet medical needs, including incurable neurodegerative diseases. However, several bottlenecks hinder CRTs, especially the needs for improved cell manufacturing processes and enhanced cell survival and integration after implantation. Engineering synthetic biomaterials that present biological signals to support cell expansion, differentiation, survival, and/or integration may help overcome […]
Macaca mulatta as advanced model for predictive preclinical testing of engineered cardiac autografts and allografts
Heart disease is the number one cause of death in the US. Heart muscle injured during a heart attack does not regenerate, and the resulting damage leads to heart failure, which inflicts almost 6 million people in the US alone. Recently, several studies have shown that direct injection of stem cell-derived heart cells may offer […]
A Chromatin Context Tool for Predicting iPS Lineage Predisposition and Tissue Graftability
Induced pluripotent stem (iPS) cells are cells derived from skin that closely resemble embryonic stem (ES) cells and can be coaxed into many different types of cells such as nerve cells, heart cells, liver cells, and also back to skin cells. One major bottleneck in the field is our ability to coax the cells into […]
A suite of engineered human pluripotent stem cell lines to facilitate the generation of hematopoietic stem cells
Our goal is to develop tools that address major bottlenecks that have prevented the generation of blood forming stem cells in culture for therapeutic use. To help overcome these bottlenecks, we will generate a suite of human embryonic stem cell reporter lines that can be used to monitor key milestones in blood stem cell development. […]
Small molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use
Tens of thousands of patients need bone marrow transplants (BMT) every year, some for bone marrow (BM) cancers and some for inherited diseases such as sickle cell anemia and thalassemia, but many lack a BM donor. African Americans, Asian Americans, and people of Hispanic descent are more likely than others to lack a stem cell […]
Identification and isolation of transplantable human hematopoietic stem cells from pluripotent cell lines; two steps from primitive hematopoiesis to transplantable definitive cells, and non-toxic conditioning of hosts for hematopoeitic stem cell transp…
A goal of stem-cell therapy is to transplant into a patient “tissue-specific” stem cells, which can regenerate a particular type of healthy tissue (e.g., heart or blood cells). A major obstacle to this goal is obtaining tissue-specific stem cells that (1) are available in sufficient numbers; and (2) will not be rejected by the recipient. […]
A small molecule tool for reducing the malignant potential in reprogramming human iPSCs and ESCs
This research project aims to solve a key bottleneck in the use of differentiated human embryonic stem cells and induced pluripotent stem cells for the regeneration and replacement of diseased or damaged tissues. This bottleneck is the potential of unintended transplants containing failed-to-differentiate stem cells developing into benign growths called teratomas, or worse, malignant teratocarcinomas. […]
Development of a clinical-grade extracorporeal liver support system using human induced pluripotent stem cell-derived hepatic cells
Liver failure is the fourth leading cause of adult death in California. Because liver cells can regenerate, some patients with liver failure could be saved without having to undergo organ transplantation if their liver function could be supported temporarily. Here, we propose to develop a device to support these patients called the “extracorporeal liver support […]
User-friendly predictive molecular diagnostic assays for quality control of stem cell derivatives for transplantation and drug discovery
Three years ago, with help from CIRM funding, we developed an assay. This is a genomics-base diagnostic assay, similar to those now used for diagnosing cancers; but in our case, it is designed to analyze human ES and iPS cells. The assay is very simple to use; researchers use microarrays to profile the genes that […]
Development of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases
Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Paralysis and in many cases death may result from a loss of motor neurons. No effective treatments are available for these patients. […]