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.
Want to see the full answer?
Check out a sample textbook solutionChapter ST Solutions
Concepts of Genetics (12th Edition)
- The acquired antibiotic resistance genes encode what types of proteins that can inhibit the function of the antibiotic? list twoarrow_forwardWith regard to the CRISPR-Cas system that defends bacteriaagainst bacteriophage attack, what happens during the adaptation,expression, and interference phases? When a bacterium is exposed toa particular bacteriophage, is the adaptation phase always necessary?arrow_forwardHow can we be sure that the insertion of the therapeutic gene does not harm some other necessary cell function?arrow_forward
- What does MEGA stand for and what is this assay used to determine? How to do perform the assay?arrow_forwardWhat mechanism of antibiotic resistance does the NDM gene codefor?arrow_forwardrs1800092 is a single nucleotide variant identifier for a variant in the CFTR gene. What type of variant is it? What is the amino acids change? Is it pathogenic?arrow_forward
- What is double-knockout animals (DKOs) ?arrow_forwardIn selecting target cells to receive a transferred gene in gene therapy, what factors do you think would have to be taken into account? Explain your answer.arrow_forwardwhich is the delivery techniques can bbe used to best transfer recombinant plasmids into E.coli cells?arrow_forward
- Do the antibiotic-resistant mutants arise in response to the presence of the antibiotic?arrow_forwardIf a vector containing the desired gene X is inserted into a microorganism, for instance, A.carbornarious, and the protoplast transformation resulted in 9 gene X transformants, how to determine which transformant has the highest titer of gene X?arrow_forwardTo understand the genes responsible for growth and infectivity in a disease-causing bacterial strain, you perform chemical mutagenesis on a culture of these bacteria. In the course of your investigation into the properties of the resulting mutants, you identify a set of mutant bacteria that is still viable but their virulence is significantly impaired. How might these mutants be useful for vaccine development?arrow_forward
- Human Heredity: Principles and Issues (MindTap Co...BiologyISBN:9781305251052Author:Michael CummingsPublisher:Cengage Learning