Genes and Gene Therapy

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.

National Human Genome Research Institute

Children

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

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 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

Teenagers

• Basics on Genes and Genetic Disorders Nemours Foundation

Reference Desk

• Biological Pathways National Human Genome Research Institute
• Chromosomes National Human Genome Research Institute
• DNA Microarray Technology National Human Genome Research Institute
• Genetic Testing Registry National Center for Biotechnology Information
• Genomics and Precision Health Weekly Update Centers for Disease Control and Prevention
• Talking Glossary of Genetic Terms National Human Genome Research Institute
• Transcriptome National Human Genome Research Institute

Related Issues

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

Clinical Trials

• ClinicalTrials.gov: Gene Therapy National Institutes of Health

Specifics

• Disease Treatments American Society of Gene & Cell Therapy

NIH MedlinePlus Magazine

• Gene Therapies are a Promising Path to Treating Rare Diseases
• Tesha Samuels Advocates for Others After Living with Sickle Cell Disease

Start Here

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

Encyclopedia

• Genes Medical Encyclopedia
• Genetics Medical Encyclopedia

Find an Expert

• National Human Genome Research Institute National Human Genome Research Institute

Journal Articles

• Gene therapy