Summary
Treating disease by introducing, repairing or replacing faulty genes in a patient's cells.
In depth
Approaches: gene replacement, gene silencing and gene editing, delivered in vivo or ex vivo. Approved examples: Luxturna (RPE65 retinal disease), Zolgensma (SMA) and CAR-T therapies. Risks include immune responses and insertional mutagenesis.
Vectors must reach target cells and avoid immune attack. Somatic therapy affects only the patient; germline changes would be inherited and are widely restricted. Costs can exceed a million dollars per treatment.
Key idea: gene therapy treats the cause of a genetic disease, not just symptoms.
Gene therapy treats disease by changing the genetic instructions in a patient's cells. Approaches include adding a working gene copy, silencing a harmful gene, editing the mutation, or equipping cells with new functions, as in CAR-T therapy for leukaemia.
It can be done ex vivo (cells modified outside the body and returned) or in vivo (vector given directly). Challenges include delivery, immune responses, insertional mutagenesis and cost. Early trials in the 1990s had serious setbacks, but approved treatments now exist for inherited blindness, spinal muscular atrophy and sickle cell disease.
Example: modified stem cells restoring immune function in children with SCID.
Key idea: somatic therapy affects only the patient, not their offspring.
Frequently asked questions
What is Gene Therapy?
Treating disease by introducing, repairing or replacing faulty genes in a patient's cells.
Which area of biology covers Gene Therapy, and how advanced is it?
Gene Therapy is one of the 56 topics in Biotechnology & Genomics. It is rated Beginner, so it suits learners who are starting out in biology. Related topics in this area include Stem Cell Therapy, Gene Therapy Vectors, Mitochondrial Replacement Therapy.
Can you give an example of Gene Therapy?
The 1990 trial for ADA-SCID was the first.
What should I learn before Gene Therapy?
Gene Therapy is a Beginner topic, so no earlier topic is required. Other Beginner topics in Biotechnology & Genomics that pair well with it include Biotechnology, Genetic Engineering, Selective Breeding.
What topics are related to Gene Therapy?
The closest topics to Gene Therapy are Stem Cell Therapy, Gene Therapy Vectors, Mitochondrial Replacement Therapy, Transgenic Organisms. Comparing them side by side is a good way to see how the ideas connect.
How can I check that I understand Gene Therapy?
Read the summary and the in-depth section, then use the quick check on the interactive lesson for Gene Therapy. To practise the whole of Biotechnology & Genomics, use the Biotechnology & Genomics quiz.
How is Gene Therapy different from Stem Cell Therapy?
Gene Therapy: Treating disease by introducing, repairing or replacing faulty genes in a patient's cells. Stem Cell Therapy: Using stem cells to replace or repair damaged tissue, such as in bone marrow transplants. Read both side by side and note what each one explains.
How should I study Gene Therapy step by step?
First, read the summary and write the definition in your own words. Second, read the in-depth section and note any example. Third, compare it with Stem Cell Therapy and explain the difference aloud. Finally, take the Biotechnology & Genomics quiz and come back to this page for any question you miss.