Which of the following diseases would be most amenable to gene therapy using ex vivo modification of hematopoietic stem cells?
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Correct answer: Inherited blood disorders such as β-thalassemia
Inherited blood disorders are well suited to ex vivo gene therapy: hematopoietic stem cells are removed, genetically corrected in culture, and reinfused to restore normal blood cell production.
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More Gene therapy basics questions
- What is one ethical concern unique to germline gene therapy (modifying sperm, eggs, or embryos) as opposed to somatic cell gene therapy?
- Which regulatory challenge is commonly associated with clinical application of gene therapy?
- Which of the following best describes why a “perfect vector” for gene therapy does not yet exist?
- How can gene therapy address diseases caused by a dominant negative mutation (where a mutant gene product interferes with normal function)?
- Which statement about non-viral gene delivery systems is correct compared to viral vectors?
- Which gene therapy approach could potentially treat a recessive loss-of-function genetic disease by restoring missing protein function?