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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.
Genetics Home Reference
Related Issues
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Brief Guide to Genomics: DNA, Genes and Genomes
(National Human Genome Research Institute) Also in Spanish
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Epigenomics
(National Human Genome Research Institute) Also in Spanish
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Frequently Asked Questions about Pharmacogenomics
(National Human Genome Research Institute) Also in Spanish
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Genetics, Disease Prevention and Treatment
(National Human Genome Research Institute)
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MedlinePlus Genetics
(National Library of Medicine)
- Personalized Medicine and Pharmacogenomics (Mayo Foundation for Medical Education and Research) Also in Spanish
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Studying Genes
(National Institute of General Medical Sciences)
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What are Whole Exome Sequencing and Whole Genome Sequencing?
(National Library of Medicine)
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What Is Genetic Ancestry Testing?
(National Library of Medicine)
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What Is Pharmacogenomics?
(National Institute of General Medical Sciences)
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What Is the Epigenome?
(National Library of Medicine)
Specifics
- Disease Treatments (American Society of Gene & Cell Therapy)
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Personalized Medicine: Matching Treatments to Your Genes
(National Institutes of Health)
Statistics and Research
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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 Launches New Collaboration to Develop Gene-Based Cures for Sickle Cell Disease and HIV on Global Scale (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
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ClinicalTrials.gov: Gene Therapy
(National Institutes of Health)
Journal Articles References and abstracts from MEDLINE/PubMed (National Library of Medicine)
- Article: CRISPR/Cas13: A potential therapeutic option of COVID-19.
- Article: Interrupting sequence variants and age of onset in Huntington's disease: clinical...
- Article: Potential use of noncoding RNAs and innovative therapeutic strategies to target...
- Genes and Gene Therapy -- see more articles
- Gene mapping -- see more articles
Reference Desk
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Biological Pathways
(National Human Genome Research Institute) Also in Spanish
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Chromosomes
(National Human Genome Research Institute) Also in Spanish
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DNA Microarray Technology
(National Human Genome Research Institute) Also in Spanish
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Genetic Testing Registry
(National Center for Biotechnology Information)
- Genomics and Health Impact Update (Centers for Disease Control and Prevention)
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Talking Glossary of Genetic Terms
(National Human Genome Research Institute) Also in Spanish
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Transcriptome
(National Human Genome Research Institute) Also in Spanish
Find an Expert
Children
- All about Genetics (For Parents) (Nemours Foundation) Also in Spanish
- What Is a Gene? (For Kids) (Nemours Foundation) Also in Spanish
Teenagers
- Basics on Genes and Genetic Disorders (Nemours Foundation) Also in Spanish