Explain how gene therapy was used to treat SCID in a patient

Biology Today and Tomorrow without Physiology (MindTap Course List)
5th Edition
ISBN:9781305117396
Author:Cecie Starr, Christine Evers, Lisa Starr
Publisher:Cecie Starr, Christine Evers, Lisa Starr
Chapter10: Biotechnology
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Explain how gene therapy was used to treat SCID in a patient 

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Step 1

Severe Combined Immunodeficiency Disease (SCID) is a rare genetic disorder characterized by the malfunction of T cells and B cells caused by various genetic mutations in the gene that codes for Adenosine Deaminase (ADA). Deficiency of ADA can prevent the body from producing the white blood cells (leukocytes) that are required to fight infections.

Step 2

Gene therapy is a method of molecular medicine based on the addition of a corrected copy of a gene to the somatic cells of an individual to cure or to alleviate the impacts of the disease.

Gene therapy is a promising treatment for ADA-SCID. During gene therapy, the retrovirus is genetically engineered to contain the normal human ADA gene. A retrovirus can transfer its DNA into normal eukaryotic cells through the process of transfection.

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