How gene therapy can treat diseases
How gene therapy can treat diseases
Gene therapy seeks to repair genetic mutations through the introduction of healthy, working genes.
Encyclopædia Britannica, Inc.
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Encyclopædia Britannica, Inc.Gene therapy seeks to repair genetic mutations through the introduction of healthy, working genes.
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Encyclopædia Britannica, Inc.The cell cycle is an ordered sequence of events that prepares a cell for the process of cell division.
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Encyclopædia Britannica, Inc.Each species has a unique set of chromosomes. These chromosomes, together with mitochondrial DNA, make up an organism's genome.
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Encyclopædia Britannica, Inc.Learn about the difference between prokaryotes and eukaryotes.
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Encyclopædia Britannica, Inc.The terms haploid and diploid refer to the number of chromosomes in each cell.
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Science in Seconds (www.scienceinseconds.com) (A Britannica Publishing Partner)DNA origami, developed by American computer scientist and bioengineer Paul Rothemund, involves folding DNA to create various shapes and structures, which may be of use to scientific investigations in a wide range of fields.
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© American Chemical Society (A Britannica Publishing Partner)CRISPR and genetically modified humans.
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Encyclopædia Britannica, Inc.The animated structure of a DNA molecule. Deoxyribose sugar molecules and phosphate molecules form the outer edges of the DNA double helix, and base pairs bind the two strands to one another.
News •
Gene therapy advances as scientists guide jumping DNA to target faulty genes
• Dec. 18, 2025, 3:18 AM ET (Medical Xpress)
South Florida teen among first to receive gene therapy to treat sickle cell anemia
• Dec. 15, 2025, 11:12 PM ET (CBS)
'Ready-made' T-cell gene therapy tackles 'incurable' T-cell leukemia
• Dec. 8, 2025, 10:40 AM ET (Medical Xpress)
Gene Therapy Offer a Cure for Babies With the Deadly ‘Bubble Boy Disease’
• Nov. 27, 2025, 3:10 AM ET (New York Times)
Transcript
NARRATOR: Genes carry the hereditary information responsible for the distinct characteristics of every individual. They are composed of DNA, and it is the sequence of bases along a strand of DNA that determines the genetic code. Disruptions of the number or order of bases in a gene are known as mutations. If severe enough, these mutations can cause death or diseases such as cancer and cystic fibrosis.
Gene therapy seeks to treat or prevent genetic disease by introducing a normal, working gene into an individual's genome. This new gene may serve to replace the mutated gene. The transformed cells then reproduce and generate normal gene product, thereby reducing the symptoms of disease or eliminating the disease altogether. If, however, the new gene replaces a working gene instead of the defective one, a new mutation may arise.
Another approach to gene therapy involves the introduction of a new gene that does not replace the mutation but rather aids the body in fighting the disease. This new gene targets the damaged cells while leaving the healthy ones unharmed.
Gene therapy seeks to treat or prevent genetic disease by introducing a normal, working gene into an individual's genome. This new gene may serve to replace the mutated gene. The transformed cells then reproduce and generate normal gene product, thereby reducing the symptoms of disease or eliminating the disease altogether. If, however, the new gene replaces a working gene instead of the defective one, a new mutation may arise.
Another approach to gene therapy involves the introduction of a new gene that does not replace the mutation but rather aids the body in fighting the disease. This new gene targets the damaged cells while leaving the healthy ones unharmed.
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