Concept explainers
To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.
Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.
To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.
Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.
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Concepts of Genetics (12th Edition)
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- Human Heredity: Principles and Issues (MindTap Co...BiologyISBN:9781305251052Author:Michael CummingsPublisher:Cengage Learning