Genes and Gene Therapy

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• Clinical Trials
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Summary

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.

Start Here

• Gene Therapy (Mayo Foundation for Medical Education and Research) Also in Spanish
• Genetics Basics: Six Things You Should Know (American Society of Human Genetics)
• How Gene Therapy Can Cure or Treat Diseases (Food and Drug Administration) Also in Spanish
• What Are Genetic Therapies? (National Heart, Lung, and Blood Institute) Also in Spanish

Related Issues

• Brief Guide to Genomics: DNA, Genes and Genomes (National Human Genome Research Institute) Also in Spanish
• Epigenomics (National Human Genome Research Institute) Also in Spanish
• Frequently Asked Questions about Pharmacogenomics (National Human Genome Research Institute) Also in Spanish
• Genetics, Disease Prevention and Treatment (National Human Genome Research Institute)
• Genetics: MedlinePlus Genetics (National Library of Medicine) Also in Spanish
• What are whole exome sequencing and whole genome sequencing?: MedlinePlus Genetics (National Library of Medicine) Also in Spanish
• What is epigenetics?: MedlinePlus Genetics (National Library of Medicine) Also in Spanish
• What is genetic ancestry testing?: MedlinePlus Genetics (National Library of Medicine) Also in Spanish
• What is Pharmacology? (National Institute of General Medical Sciences)

Specifics

• Disease Treatments (American Society of Gene & Cell Therapy)

Statistics and Research

• All about the Human Genome Project (HGP) (National Human Genome Research Institute)
• Coming of Age of Gene Therapy: A Review of the Past and Path Forward (National Institutes of Health)
• NIH Researchers Create New Viral Vector for Improved Gene Therapy in Sickle Cell Disease (National Heart, Lung, and Blood Institute)

Clinical Trials

• ClinicalTrials.gov: Gene Therapy (National Institutes of Health)

Journal Articles References and abstracts from MEDLINE/PubMed (National Library of Medicine)

• Article: Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and...
• Article: AAV-NDI1 Therapy Provides Significant Benefit to Murine and Cellular Models of...
• Article: Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive...
• Genes and Gene Therapy -- see more articles

Reference Desk

• Biological Pathways (National Human Genome Research Institute) Also in Spanish
• Chromosomes (National Human Genome Research Institute) Also in Spanish
• DNA Microarray Technology (National Human Genome Research Institute) Also in Spanish
• Genetic Testing Registry (National Center for Biotechnology Information)
• Talking Glossary of Genetic Terms (National Human Genome Research Institute) Also in Spanish
• Transcriptome (National Human Genome Research Institute) Also in Spanish

Find an Expert

• National Human Genome Research Institute

Children

• All about Genetics (For Parents) (Nemours Foundation) Also in Spanish
• Gene Changes (Mutations) (Nemours Foundation) Also in Spanish
• What Is a Gene? (Nemours Foundation) Also in Spanish

Teenagers

• Basics on Genes and Genetic Disorders (Nemours Foundation) Also in Spanish