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.
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 Gene Therapy Vectors, Gene Drives, 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 Gene Therapy Vectors, Gene Drives, Mitochondrial Replacement Therapy, Selective Breeding. 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 Gene Therapy Vectors?
Gene Therapy: Treating disease by introducing, repairing or replacing faulty genes in a patient's cells. Gene Therapy Vectors: Viral vectors such as AAV and lentivirus deliver therapeutic genes, with safety and immune challenges. 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 Gene Therapy Vectors and explain the difference aloud. Finally, take the Biotechnology & Genomics quiz and come back to this page for any question you miss.