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  1. How does gene therapy work?: MedlinePlus Genetics

    How does gene therapy work? Gene therapy works by altering the genetic code to recover the functions of critical proteins. Proteins are the workhorses of the cell and the structural basis of the body’s tissues.

  2. How Does Gene Therapy Work? Types, Uses, Safety - Healthline

    Jan 21, 2023 · Gene therapy can treat certain genetic conditions by editing diseased cells. Learn about its uses, safety, and potential downsides here.

  3. What Is Gene Therapy? Pros, Cons & Examples - Cleveland Clinic

    Jun 17, 2025 · Gene therapy is a way to treat some genetic diseases. It works by replacing a malfunctioning gene, “turning off” a disease-causing gene or giving you a new copy of a gene.

  4. Gene therapy - Mayo Clinic

    Apr 23, 2024 · DNA controls everything from hair color and height to breathing, walking and digesting food. Genes that don't work properly can cause disease. Sometimes these genes are called …

  5. Gene Therapy - National Human Genome Research Institute

    2 days ago · Gene therapy is a technique that uses a gene (s) to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by …

  6. Gene therapy | Description, Uses, Examples, & Safety Issues - Britannica

    Nov 28, 2025 · Gene therapy, introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. Human gene therapy has been attempted on …

  7. What is Gene Therapy? - ASGCT

    Gene therapy is the use of genetic material to treat or prevent disease. The genetic material that is delivered, DNA or RNA, has instructions to change how a protein—or group of proteins—is produced …

  8. Gene Therapy Explained: Fixing DNA to Cure Disease

    May 14, 2025 · Gene therapy is a biomedical technique that uses genetic material — DNA or RNA — to treat, prevent, or potentially cure disease. It works by directly altering the genetic instructions within a …

  9. How Gene Therapy Can Cure or Treat Diseases | FDA

    Jul 28, 2022 · When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient. When gene therapy is used to modify cells outside...

  10. What Is Gene Therapy: Risks, Benefits, and More - Verywell Health

    Aug 13, 2024 · The idea behind gene therapy is to direct the body to produce healthy proteins that do not cause disease. This therapy involves the delivery of DNA or RNA. The RNA molecule is an …