Genes and Gene Therapy

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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

Start Here

Frequently Asked Questions about Genetic and Genomic Science (National Human Genome Research Institute) Also in Spanish
Gene Therapy (Mayo Foundation for Medical Education and Research) Also in Spanish
New Genetics (National Institute of General Medical Sciences)
Six Things Everyone Should Know About Genetics (American Society of Human Genetics)

Related Issues

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)
Personalized Medicine and Pharmacogenomics (Mayo Foundation for Medical Education and Research) Also in Spanish
Studying Genes (National Institute of General Medical Sciences)
What are Whole Exome Sequencing and Whole Genome Sequencing? (National Library of Medicine)
What Is Genetic Ancestry Testing? (National Library of Medicine)
What Is the Epigenome? (National Library of Medicine)

Specifics

Personalized Medicine: Matching Treatments to Your Genes (National Institutes of Health)

Games

Michael's Inside Scoop: Genetics (Centers for Disease Control and Prevention)

Statistics and Research

All about the Human Genome Project (HGP) (National Human Genome Research Institute)
Genetics by the Numbers (National Institute of General Medical Sciences)

Clinical Trials

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

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

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)
Genomics and Health Impact Update (Centers for Disease Control and Prevention)
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 Therapy and Children (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

Basics

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Research

Resources

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