Project Objective: Tool/Resources/Bottleneck
Generation of functional cells and organs from iPSCs
The development of induced pluripotent stem cell (iPSC) technology may be the most important advance in stem cell biology for the future of medicine. This technology allows one to generate a patient’s own pluripotent stem cells (PSCs) from skin or blood cells. iPSCs can then be reprogrammed to multiply and produce high quality mature cells […]
Genetic Encoding Novel Amino Acids in Embryonic Stem Cells for Molecular Understanding of Differentiation to Dopamine Neurons
Embryonic stem cells have the capacity to self-renew and differentiate into other cell types. Understanding how this is regulated on the molecular level would enable us to manipulate the process and guide stem cells to generate specific types of cells for safe transplantation. However, complex networks of intracellular cofactors and external signals from the environment […]
Enhancing Survival of Embryonic Stem Cell-Derived Grafts by Induction of Immunological Tolerance
Although ESC-based therapies hold great promise for the cure of a wide diversity of degenerative diseases, rapid progress to actual human clinical trials is hindered by the lack of preclinical data for specific ESC-based therapies. I aim to move the process forward by establishing a protocol in which immune system cells are reproducibly produced from […]
Bioengineering technology for fast optical control of differentiation and function in stem cells and stem cell progeny
Embryonic stem (ES) cells potentially could provide clinically important replacement tissue for central nervous system (CNS) disease treatment, and regenerative medicine approaches involving ES cells have been suggested for common CNS disorders. But it has been difficult to produce the right kind of replacement tissues from ES cells because the “differentiation”, or cell-type specification process, […]
Addressing the Cell Purity and Identity Bottleneck Through Generation and Expansion of Clonal Human Embryonic Progenitor Cell Lines
Human embryonic stem (hES) cells and induced pluripotent (iPS) cells, such as reprogrammed skin cells, offer the potential to revolutionize medicine because they can replicate indefinitely and become virtually any cell in the body. They therefore have the potential to provide a limitless source of cells to replace cells lost to injury (spinal cord, skin […]
In Utero Model to Assess the Fate of Transplanted Human Cells for Translational Research and Pediatric Therapies
nfants with inherited blood diseases (such as sickle cell anemia, thalassemia, bleeding disorders) or other inherited metabolic disorders can be identified early in development using sophisticated diagnostic tests. Currently, the treatment for many of these childhood illnesses may include bone marrow transplantation which is complicated by: (1) the toxicity associated with chemotherapy or radiation-based regimens […]
Mouse Models for Stem Cell Therapeutic Development
Stem cells have tremendous potential for treating human diseases, as they have the unique capacity to develop into any cell type in the body and to proliferate indefinitely. The development of new therapies based on the transplantation of human stem cells (HuSC) into patients is a major focus of California researchers. A critical step prior […]
Maximizing the Safety of Induced Pluripotent Stem Cells as an Infusion Therapy: Limiting the Mutagenic Threat of Retroelement Retrotransposition during iPSC Generation, Expansion and Differentiation
The ability to convert human skin cells to induced pluripotent stem cells (IPSCs) represents a seminal break-through in stem cell biology. This advance effectively circumvents the problem of immune rejection because the patient’s own skin cells can be used to produce iPSCs. This exciting technology could accelerate treatments for a number of presently incurable diseases. […]
Ensuring the safety of cell therapy: a quality control pipeline for cell purification and validation
The clinical application of cell replacement therapy in the US is dependent on the FDA’s approval, and the primary objective of the FDA is to protect patients from unsafe drugs and procedures. The FDA has a specific mandate for human gene and cell therapy and since the unexpected deaths in early trials of gene therapy […]
Methods for detection and elimination of residual human embryonic stem cells in a differentiated cell product
Human embryonic stem cells (hESC), and other related pluripotent stem cells, have great potential as starting material for the manufacture of curative cell therapies. This is primarily for two reasons. First, by manipulating cues in their cell culture conditions, these cells can be directed to become essentially any desired human cell type (a property known […]