Technology or method
Gene editing
Making targeted changes to DNA in living cells. Newer base and prime editors change letters without cutting both strands.
Emerging researchOpen in Biotech Lab
Gene therapy
Treating disease by adding, repairing or switching off genes in a patient's cells.Source: FDASource: NHGRISource: FDA, 2023
Step 5 of 7ReadingTechnology or methodEmerging research
How it works
Gene therapy treats disease by adding, fixing or switching off a gene inside a patient's cells.
Viruses with their disease-causing genes removed can deliver a working gene. In other approaches, a patient's own cells are modified outside the body and returned, as in CAR T-cell therapy.
Approved products use adeno-associated viral vectors, lentiviral vectors or genome editing. Long-term durability, safety and very high costs are open questions.
- Used for
- Some inherited blindness
- Spinal muscular atrophy
- Certain blood cancers
- Sickle cell disease
Safety and ethics
Access and cost are major concerns, along with long-term safety monitoring.
How Gene therapy connects
Gene therapy is made possible by
Gene therapy uses
Organism or group
Virus
Nucleic acid in a protein coat. A virus cannot copy itself alone: it must infect a cell and use the cell's machinery.
Gene therapy is related to
Health condition
Sickle cell disease
An inherited blood disorder caused by a variant in the HBB gene that changes one amino acid in hemoglobin.
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Sources & further reading
The explanations on this page are our own summaries of these sources. Follow a link to read the original; if anything here disagrees with it, the original wins. How we choose and check sources.
What is Gene Therapy? (opens the original in a new tab)
U.S. Food and Drug Administration. fda.gov. U.S. Food and Drug Administration.
ReferenceGovernment sourceEducationalGene Therapy (opens the original in a new tab)
National Human Genome Research Institute. Talking Glossary of Genomic and Genetic Terms. National Human Genome Research Institute, NIH.
ReferenceGovernment sourceEducationalFDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (opens the original in a new tab)
U.S. Food and Drug Administration (2023). FDA News Release. U.S. Food and Drug Administration. Published 2023-12-08.
Approval of Casgevy (CRISPR-based) and Lyfgenia for patients 12 and older.
Regulatory noticeGovernment sourceEfficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial (opens the original in a new tab)
Russell S, Bennett J, Wellman JA, et al. (2017). The Lancet 390:849-860.
Peer-reviewedClinical trialSingle-dose gene-replacement therapy for spinal muscular atrophy (opens the original in a new tab)
Mendell JR, Al-Zaidy S, Shell R, et al. (2017). New England Journal of Medicine 377:1713-1722.
Peer-reviewedClinical trialTisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia (opens the original in a new tab)
Maude SL, Laetsch TW, Buechner J, et al. (2018). New England Journal of Medicine 378:439-448.
Peer-reviewedClinical trial