gene therapy

medicine
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Also known as: gene transfer therapy, genetic modification
Also called:
gene transfer therapy
Key People:
Fred H. Gage

News

New gene therapy reverses heart failure in large animal model Dec. 11, 2024, 12:14 AM ET (Medical Xpress)

gene therapy, introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be ...(100 of 712 words)