From NTSA case study https://static.nsta.org/case_study_docs/case_studies/cystic_fibrosis.pdf Please help with questions 2, 3 and 4 of part four 2. "The successful use of gene therapy to cure SCID syndrome (2000) is hoped to be a permanent cure for those patients because a good copy of the problem gene was inserted into the patients' blood stem cells in the bone marrow (hematopoietic stem cells). Once white blood cells enter the blood stream they have a limited life span, on the order of a few week to months. The blood stem cells are the cells that create more white blood cells to replace those that are lost. If the gene was only inserted into the circulating mature white blood cells, the patient would only be temporarily cured until those cells were used up or died." The current gene therapy approaches to cure CF involve inserting a functional CFTR gene into the mature epithelial cells of the lungs. In light of the preceding paragraph, do you think that this approach would be a "permanent" cure for CF? Explain your answer.
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From NTSA case study https://static.nsta.org/case_study_docs/case_studies/cystic_fibrosis.pdf
Please help with questions 2, 3 and 4 of part four
2. "The successful use of gene therapy to cure SCID syndrome (2000) is hoped to be a permanent cure for those patients because a good copy of the problem gene was inserted into the patients' blood stem cells in the bone marrow (hematopoietic stem cells). Once white blood cells enter the blood stream they have a limited life span, on the order of a few week to months. The blood stem cells are the cells that create more white blood cells to replace those that are lost. If the gene was only inserted into the circulating mature white blood cells, the patient would only be temporarily cured until those cells were used up or died." The current gene therapy approaches to cure CF involve inserting a functional CFTR gene into the mature epithelial cells of the lungs. In light of the preceding paragraph, do you think that this approach would be a "permanent" cure for CF? Explain your answer.
3. What level of risk should be acceptable for a patient involved in a clinical trial? In other words, under what circumstances should Nancy feel comfortable enrolling Joshua in a gene therapy clinical trial?
4. In the current clinical trials for gene therapy treatments of CF, participants must be over 12, so Joshua could not be helped by the trials that are currently being run at this time. Why might there be an age restriction such as this? Is an age restriction such as this fair?
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