Stage of Program: Translational Tool/Bottleneck
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 […]
Developing induced pluripotent stem cells into human therapeutics and disease models
Human embryonic stem cells (hESCs) can undergo unlimited self-renewal and differentiate into all the cell types in the human body, and thus hold great promise for cell replacement therapy. However, one major problem for hESC-based therapy is that the cells derived from hESCs will be rejected by the recipient and can only be tolerated under […]
Using patient-specific iPSC derived dopaminergic neurons to overcome a major bottleneck in Parkinson’s disease research and drug discovery
The goals of this study are to develop patient-specific induced pluripotent cell lines (iPSCs) from patients with Parkinson’s disease (PD) with defined mutations and sporadic forms of the disease. Recent groundbreaking discoveries allow us now to use adult human skin cells, transduce them with specific genes, and generate cells that exhibit characteristics of embryonic stem […]
Induction of immune tolerance to human embryonic stem cell-derived allografts
Human embryonic stem cells (hESCs) can undergo unlimited reproduction and retain the capability to differentiate into all cell types in the body. Therefore, as a renewable source of various cell types, hESCs hold great promise for human cell replacement therapy. Significant progress has been made in establishing the conditions to differentiate hESCs into cells of […]
Development of an immune tolerant hESC source for allogeneic cell therapy applications
Human embryonic stem cells (hESCs) are an ideal tissue source for cell replacement therapy (CRT). They have the potential for limitless self-renewal while retaining their ability to differentiate into a wide variety of cells and tissues. Since their first derivation in 1998, hESCs have been used in many studies in order to evaluate their potential […]
Development of the Theracyte Cellular Encapsulation System for Delivery of human ES Cell-derived Pancreatic Islets and Progenitors.
There are several challenges to the successful implementation of a cellular therapy for insulin dependent diabetes derived from Human Embryonic Stem Cells (hESCs). Among these are the development of functional insulin-producing cells, a clinical delivery method that eliminates the need for chronic immunosuppression, and assurance that hESC-derived tumors do not develop in the patient. We […]
Novel Separation of Stem Cell Subpopulations
The inability to separate stem cells and their differentiated progeny accurately, easily, and rapidly undermines progress in the stem cell field. Traditional separation of living cells into subpopulations relies on techniques that utilize characteristic cell surface markers, but specific markers are severely limited or lacking altogether for many stem cell populations. Without ways to discriminate […]