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
- Frequently Asked Questions about Genetic and Genomic Science (National Human Genome Research Institute)
- Gene Therapy (Mayo Foundation for Medical Education and Research)
- New Genetics (National Institute of General Medical Sciences)
- Six Things Everyone Should Know About Genetics (American Society of Human Genetics)
- Biological Therapies for Cancer (National Cancer Institute) Also in Spanish
- 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)
- Genetics Home Reference (National Library of Medicine)
- Genetics, Disease Prevention and Treatment (National Human Genome Research Institute)
- Matching Genes and Vitamins: A Personalized Plan May Be in Your Future (National Institutes of Health)
- Personalized Medicine and Pharmacogenomics (Mayo Foundation for Medical Education and Research)
- Studying Genes (National Institute of General Medical Sciences)
- What Advances Are Being Made in DNA Sequencing? (National Library of Medicine)
- What Is Genetic Ancestry Testing? (National Library of Medicine)
- What Is the Epigenome? (National Library of Medicine)
- What Role Do Epigenetics and Developmental Epigenetics Play in Health and Disease? (National Institute of Child Health and Human Development)
- Gene Therapy for Diseases and Genetic Disorders (American Society of Gene & Cell Therapy)
- Personalized Medicine: Matching Treatments to Your Genes (National Institutes of Health)
- Michael's Inside Scoop: Genetics (Centers for Disease Control and Prevention)
- ClinicalTrials.gov: Gene Therapy (National Institutes of Health)
Journal ArticlesReferences and abstracts from MEDLINE/PubMed (National Library of Medicine)
- Article: Therapy Development for the Lysosomal Storage Disease Fucosidosis using the...
- Article: Insights into the Pathogenesis and Treatment of Krabbe Disease.
- Article: CLN2 Disease (Classic Late Infantile Neuronal Ceroid Lipofuscinosis).
- Genes and Gene Therapy -- see more articles
- Gene mapping -- see more articles