Human Stem Cell Use: iPS Cell
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. […]
Autologous Retinal Pigmented Epithelial Cells Derived from Induced Pluripotent Stem Cells for the Treatment of Atrophic Age Related Macular Degeneration
The leading cause of visual loss in Americans over the age of 65 is age related macular degeneration (AMD) which occurs in both a “wet” and a “dry” form. Both forms of the disease are associated with loss of cells called retinal pigmented epithelium (RPE) which can lead to profound loss of central vision. Currently, […]
Development of a Stem Cell-based Transplantation Strategy for Treating Age-related Macular Degeneration
Age related macular degeneration (AMD) is a blinding disease of the elderly affecting nearly one in three individuals over the age of 75. Central vision is lost in AMD, severely impairing the ability to read, watch television, or drive. The epicenter of AMD is the retinal pigment epithelium (RPE), a single layer of cells in […]
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 […]
Stem Cell-Based Therapy for Cartilage Regeneration and Osteoarthritis
Arthritis is the result of degeneration of cartilage (the tissue lining the joints) and leads to pain and limitation of function. Arthritis and other rheumatic diseases are among the most common of all health conditions and are the number one cause of disability in the United States. The annual economic impact of arthritis in the […]
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 after spinal grafting of human ES-derived neural precursors
Previous clinical studies have shown that grafting of human fetal brain tissue into the CNS of adult recipients can be associated with long-term (more then 10 years) graft survival even after immunosuppression is terminated. These clinical data represent in part the scientific base for the CNS to be designated as an immune privilege site, i.e., […]
Engineering Embryonic Stem Cell Allografts for Operational Tolerance
Stem cells, like all transplants not derived from an identical twin, are subject to scrutiny by the immune system and, without medical interventions that suppress the immune system, are usually killed after transplantation. However, rare exceptions to this rule exist because a small fraction of transplant patients has been able to maintain their transplant in […]
Donor natural killer (NK) cells as “veto” cells to promote donor-specific tolerance
A major issue in the use of stem cells or in organ transplantation in general is the need to overcome graft rejection. Unfortunately, the only means currently available involves the use of systemic immunosuppression which leaves the recipient at risk for opportunistic infections. This proposal will seek to use the donor’s immune cells to prevent […]