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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.

Start Here

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

Related Issues

Specifics

  • Disease Treatments (American Society of Gene & Cell Therapy)

Statistics and Research

  • All about the Human Genome Project (HGP) From the National Institutes of Health (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

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

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

  • Article: An In Vivo Screening Model for Investigation of Pathophysiology of Human...
  • Article: Advances in Gene Therapy Techniques to Treat LRRK2 Gene Mutation.
  • Article: Lentiviral Gene Therapy for Artemis-Deficient SCID.
  • Genes and Gene Therapy -- see more articles

Reference Desk

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

Find an Expert

  • National Human Genome Research Institute From the National Institutes of Health

Children

  • All about Genetics (For Parents) (Nemours Foundation) Also in Spanish
  • Gene Changes (Mutations) (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

The information on this site should not be used as a substitute for professional medical care or advice. Contact a health care provider if you have questions about your health.

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