Curing Hematological Diseases
The primary aim of this project is to develop treatments for incurable diseases of the blood and immune system. X-linked Severe Combined Immunodeficiency (X-SCID) and Fanconi anemia (FA) are two…
Developmental Candidates for Cell-Based Therapies for Parkinson’s Disease (PD)
Parkinson's Disease (PD) is a devastating disorder, stealing vitality from vibrant, productive adults & draining our health care dollars. It is also an excellent model for studying other neurodegenerative conditions.…
Enhancing healing via Wnt-protein mediated activation of endogenous stem cells
All adult tissues contain stem cells. Some tissues, like bone marrow and skin, harbor more adult stem cells; other tissues, like muscle, have fewer. When a tissue or organ is…
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…
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…
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…
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…
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…
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.…
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…
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