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- Services and providers for Genes and Gene Therapy in the U.S.
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National Institutes of Health
- The primary NIH organization for research on Genes and Gene Therapy is the National Human Genome Research Institute
Genes and Gene Therapy
Genes are the building blocks of inheritance. Passed from parent to child, they contain instructions for making proteins. If genes don't produce the right proteins or don't produce them correctly, a child can have a genetic disorder.
Gene therapy is an experimental technique that uses genes to treat or prevent disease. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include
- Swapping an abnormal gene for a normal one
- Repairing an abnormal gene
- Altering the degree to which a gene is turned on or off
Although there is much hope for gene therapy, it is still experimental.
Genetics Home Reference
Start Here
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Frequently Asked Questions about Genetic and Genomic Science
(National Human Genome Research Institute)
- Genetics Home Reference: What is gene therapy?(National Library of Medicine)
- JAMA Patient Page: Genetics: The Basics(American Medical Association)
| Basics | Learn More | Multimedia & Cool Tools |
|---|---|---|
| Research | Reference Shelf | For You |
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Overviews
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From Genes to Personalized Medicine(National Institutes of Health) - Links to PDF
- Gene Therapy(Dept. of Energy)
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Genes -- What We Knew, Know, and Hope to Learn(National Institute of General Medical Sciences)
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New Genetics(National Institute of General Medical Sciences)
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From Genes to Personalized Medicine
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Latest News
- Colon Cancer Oncogene Discovered(09/14/2008, HealthDay)
- Gene-Transfer Proves Safe for Vision Problem(09/12/2008, HealthDay)
- Broccoli Compound May Help COPD Patients(09/12/2008, HealthDay)
- More News on Genes and Gene Therapy
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Related Issues
- FDA 101: Human Gene Therapy(Food and Drug Administration)
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Gene Therapy for Cancer: Questions and Answers(National Cancer Institute)
- Gene Therapy: Cancer Research Topic Proves Challenging, but Promising(Mayo Foundation for Medical Education and Research)
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Genetics Home Reference(National Library of Medicine)
- Genomics and Medical Devices: A New Paradigm for Health Care(Food and Drug Administration)
- Genomics and Personalized Medicine(Food and Drug Administration)
- How FDA Advances Personalized Medicine(Food and Drug Administration)
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Human Genetic Variation(National Institute of General Medical Sciences)
- Metabolomics: Working toward Personalized Medicine(Food and Drug Administration)
- Pharmacogenomics: When Drug Treatment Becomes Personalized Medicine(Mayo Foundation for Medical Education and Research)
- Proteomics: Moving Beyond the Human Genome(Food and Drug Administration)
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Games
- Play the DNA - The Double Helix Game(Nobel Foundation) - Requires Flash Player
- Play the Genetic Code Game(Nobel Foundation) - Requires Flash Player
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Anatomy/Physiology
- Gene-Environment Interaction Fact Sheet(Centers for Disease Control and Prevention)
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Clinical Trials
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ClinicalTrials.gov: Genes(National Institutes of Health)
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ClinicalTrials.gov: Gene Therapy(National Institutes of Health)
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ClinicalTrials.gov: Genes
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Research
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All about the Human Genome Project (HGP)(National Human Genome Research Institute)
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Cell-Signaling System Helps Explain How Skin Develops into Different Types(National Institute of Arthritis and Musculoskeletal and Skin Diseases)
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Genes or Environment? Epigenetics Sheds Light on Debate(National Institutes of Health)
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Genes, Behavior, the Environment and Health(National Institutes of Health) - Links to PDF
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Medicines for You: Studying How Your Genes Can Make a Difference(National Institute of General Medical Sciences)
Also available in Spanish
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Pharmacogenetics(National Institute of General Medical Sciences)
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All about the Human Genome Project (HGP)
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Journal Articles
References and abstracts from MEDLINE/PubMed (National Library of Medicine)
- Article: Toward gene therapy of endometriosis: transductional and transcriptional targeting of...
- Article: Optimizing exon skipping therapies for DMD.
- Article: Patient page. Gene therapy and muscular dystrophies.
- Genes and Gene Therapy -- see more articles
- Gene mapping -- see more articles
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Dictionaries/Glossaries
- "-OMICS" Glossary: A Selection of Important Terms and Concepts Defined(Food and Drug Administration)
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Talking Glossary of Genetic Terms(National Human Genome Research Institute) - Requires media player
Also available in Spanish
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Directories
- Laboratory Directory-GeneReviews(University of Washington)
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Organizations
- GeneTests(University of Washington)
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National Human Genome Research Institute
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Newsletters/Print Publications
- Genomics & Health Weekly Update(Centers for Disease Control and Prevention)
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Law and Policy
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Issues in Genetics and Health(National Human Genome Research Institute)
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Policy and Legislation Database(National Human Genome Research Institute)
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Issues in Genetics and Health
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Children
- Gene Therapy and Children(Nemours Foundation)
- What Is a Gene?(Nemours Foundation)
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You and Your Genes: Making It in a Tough Environment(National Institute of Environmental Health Sciences) - Links to PDF
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Teenagers
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Basics on Genes and Genetic Disorders(Nemours Foundation)
Also available in Spanish
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Basics on Genes and Genetic Disorders(Nemours Foundation)
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Date last updated: 15 September 2008 Topic last reviewed: 31 July 2008 |