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When gene targetting involving gene amplification is attempted in an individual's tissue to treat disease, it is known as:
The introduction of normal, functional genes into human cells or tissues to correct hereditary or acquired disease defects is termed Gene Therapy (e.g., ADA deficiency treatment in 1990)! Match Option (2)!
Gene therapy encompasses medical interventions designed to correct genetic defects diagnosed in a child or embryo:
In 1990, the first clinical gene therapy was administered to a 4-year-old girl suffering from adenosine deaminase () deficiency. Lymphocytes were harvested from the patient's blood, transformed in vitro using a retroviral vector carrying functional for , and re-infused into her body.
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Biopiracy is the unethical use of bio-resources by multinational companies without authorization or compensatory payment to indigenous communities.
Gene therapy is the delivery of normal functional genes into patient tissues to treat diseases caused by defective alleles.
Molecular diagnosis refers to diagnostic identification of pathogens/mutations (e.g., PCR, ELISA), not therapeutic gene insertion.
Safety testing involves toxicology assays in transgenic animals (e.g., polio vaccine testing in mice), not therapeutic intervention.
The stem describes an attempt "to treat disease". Diagnosis detects disease; Therapy cures or treats disease.
"Gene therapy is a collection of methods that allows correction of a gene defect that has been diagnosed in a child/embryo."
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