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Gene Therapy

In gene therapy, genetic material that provides a missing or necessary protein, or causes a clinically-relevant biochemical process, is introduced into an organ for a therapeutic effect. For gene-based therapies (specifically, those using DNA sequences), it is critical that the desired gene be introduced into organ stem cells in order to achieve long-term expression and therapeutic effect. Although techniques for delivering the therapeutic DNA have been greatly improved since the first gene therapy protocol almost 10 years ago, there are as yet no bona fide successes. Besides delivery problems, loss of expression or insufficient expression is an important limiting factor in successful application of gene therapy and could be overcome by transferring genes into stem cells (which presumably will then differentiate and target correctly).

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Go to Genetics Topic Index
Stem Cell Research and Applications: Monitoring the Frontiers of Biomedical Research
Preface
Findings and Recommendations
The Science of Stem Cell Research and Potential Therapies
     Current Status of Human Stem Cell Research
     Sources and Characteristics of Human Stem Cells
        Human Embryonic Stem Cells
        Human Embryonic Germ Cells.
        Human Adult Stem Cells
     The Clinical Potentials for Stem Cell Products
     Some Examples of Treatments for Major Diseases
        Type 1 Diabetes in Children
        Nervous System Diseases
        Primary Immunodeficiency Diseases
        Diseases of Bone and Cartilage
        Cancer
     Uses in Research
        A New Window on Human Developmental Biology
        Models of Human Disease that are Constrained by Current Animal and Cell Culture Models
        Transplantation
        Gene Therapy
Spiritual and Religious Contexts
Ethical Concerns
     The Moral Status of Human Stem Cells
     Moral Issues Surrounding the Sources of Stem Cells
Sources of Stem Cells and Guidelines for Use
Justice Considerations
Funding
Oversight and Accountability
     Private Sector Oversight
     Intellectual Property Considerations
     Public Sector Oversight
Conclusion
Appendix I: Working Group Members
Appendix II: Staff
Appendix III: About AAAS and ICS

Source:

Audrey R. Chapman, Ph.D., Mark S. Frankel, Ph.D., and Michele S. Garfinkel, Ph.D. for the American Association for the Advancement of Science and The Institute for Civil Society

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