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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
• Gene Therapy Approaches (American Society of Gene & Cell Therapy)
• How does gene therapy work?: MedlinePlus Genetics (National Library of Medicine)
• 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
• What is a gene?: MedlinePlus Genetics (National Library of Medicine)
• What is gene therapy?: MedlinePlus Genetics (National Library of Medicine)

Related Issues

• Brief Guide to Genomics (National Human Genome Research Institute) Also in Spanish
• Epigenomics (National Human Genome Research Institute) Also in Spanish
• Genetics, Disease Prevention and Treatment FAQ (National Human Genome Research Institute)
• Genetics: MedlinePlus Genetics (National Library of Medicine) Also in Spanish
• Pharmacogenomics (National Human Genome Research Institute)
• 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 Genetics? (National Institute of General Medical Sciences)
• What is Pharmacology? (National Institute of General Medical Sciences)

Specifics

• Condition Treatments (American Society of Gene & Cell Therapy)
• What are CAR T cell therapy, RNA therapy, and other genetic therapies?: MedlinePlus Genetics (National Library of Medicine)
• What are genome editing and CRISPR-Cas9?: MedlinePlus Genetics (National Library of Medicine)

Statistics and Research

• Gene Circuits Enable More Precise Control of Gene Therapy (Massachusetts Institute of Technology)
• Human Genome Project (HGP) (National Human Genome Research Institute)
• Infant with Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment (National Institutes of Health)

Clinical Trials

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

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

• Article: Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
• Article: A phase I/II trial of WT1-specific TCR gene therapy for patients...
• Article: OVATION-2: A randomized phase I/II study evaluating the safety and efficacy...
• 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 Genomic and Genetic Terms (National Human Genome Research Institute) Also in Spanish
• Transcriptome (National Human Genome Research Institute) Also in Spanish

Find an Expert

• Gene Therapy Centers (American Society of Gene & Cell Therapy)
• National Human Genome Research Institute

Children

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

Teenagers

• Genes and Genetics (Nemours Foundation) Also in Spanish