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What is gene therapy?
Reviewed by Medicinsk Expert•Last updated: 2026-10-22•Therapy

What is gene therapy?

Gene therapy is a medical approach that treats or prevents disease by correcting underlying genetic problems rather than relying solely on drugs or surgery.

Gene Transfer and Addition

The earliest method of gene therapy, often called gene transfer or gene addition, was developed to:

  • Introduce a new gene into cells to help fight a disease.
  • Introduce a non-faulty copy of a gene to stand in for the altered copy causing disease.

Genome Editing

A newer technique, called genome editing (such as CRISPR-Cas9), uses a different approach. Instead of introducing new genetic material, it introduces molecular tools to change the existing DNA in the cell. Genome editing is being studied to:

  • Fix a genetic alteration underlying a disorder so the gene can function properly.
  • Turn on a gene to help fight a disease.
  • Turn off a gene that is functioning improperly.
  • Remove a piece of DNA that is impairing gene function and causing disease.

Current Status

Gene therapies are currently used to treat a small number of diseases, including the eye disorder Leber congenital amaurosis and the muscle disorder spinal muscular atrophy. Many more therapies are undergoing research to ensure they are safe and effective, and genome editing remains a promising technique under study for future use in humans.

Frequently Asked Questions

Currently, gene therapies are used to treat a small number of specific diseases. Examples mentioned include the eye disorder Leber congenital amaurosis and the muscle disorder spinal muscular atrophy. Many more therapies are actively being researched for safety and effectiveness.
While genome editing techniques like CRISPR-Cas9 are very promising, the context states that genome editing remains a promising technique under study for future use in humans. It is not yet widely used for treatment, unlike gene transfer/addition which has a few approved therapies.
Gene therapy can address genetic problems in a few ways. It can introduce a new gene or a non-faulty copy of a gene to help fight a disease or replace an altered gene. Newer techniques like genome editing can fix existing genetic alterations, turn genes on or off, or remove problematic pieces of DNA to restore proper function.
Gene therapy takes a fundamentally different approach by directly correcting the underlying genetic problems that cause a disease. Unlike drugs that often manage symptoms or surgery that addresses physical issues, gene therapy aims to modify the genetic instructions within cells to prevent or treat the disease at its source.
Source: MedlinePlus, National Library of Medicine

MedlinePlus brings together authoritative health information from the National Library of Medicine (NLM), the National Institutes of Health (NIH), and other government agencies and health-related organizations.

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