Applications
Tissue Repair and Replacement
A number of diseases are characterized by the death of specific cell populations that do not regenerate themselves. Diseases such as neurodegenerative disorders, paralysis and Type I diabetes affect millions of people in the United States alone, and currently no effective long-term therapies are available. The transplantation of human neural stem cells could potentially provide a way to repair tissue damaged by such diseases and injuries.
Drug Discovery and Screening
Stem cells are a rich source of information for the drug discovery process, as they naturally produce many factors related to cell growth and differentiation.
Functional Genomics
The ability to study which genes are turned on and off early in development, and what sorts of environmental signals contribute to the regulation of those genes, could provide valuable insights into the functional genomics of developing cells.
Correction of Genetic Disorders
One of the problems in achieving effective gene therapy has been the inability to achieve the truly long-term gene expression necessary for successful treatment of genetic disorders. The therapeutic value of stem cells arises from their ability to self-renew and become the cell types characteristic of the organ into which they are transplanted, enabling the long-term integration of new tissue into damaged organs. This ability to integrate and generate new cells within an organ makes stem cells prime candidates as vehicles to deliver gene therapy.
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