(A) A lab mouse suffers from Cystic Fibrosis, which is caused by a nonsense muta
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(A) A lab mouse suffers from Cystic Fibrosis, which is caused by a nonsense mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. You decided to perform gene therapy to cure this disease for this CF mouse. There are two alternative experimental designs (only one is correct). The first is to introduce a wild type CFTR gene into the CF mice. The second is to introduce CAS9 and sgRNA into the CF mice to knockout the CFTR gene. Which one would you choose and why? (B) One of your friends has Cystic Fibrosis and is preparing for gene therapy treatment. He is provided with two options in the viral vectors: the "retroviral vector" and the "Adeno-associated viral vector". He comes to you for advice. Could you tell him one limitation for "retroviral vector" and one limitation for "Adeno-associated viral vector"?|Explanation / Answer
1. to ibtroduce CAS9 and sg RNA into CF mice as wild type CFTR does not reach the apical membrane as does transgene product, hence it is convenient to use CAS9 sgRNA into apical CFTR
2.long term survival on usage of CAS and sg RNA into mice has been documented
B. Retrovoral gene theraphy is indicated as
1. absence of coxackie adenovirus recoptors on apicqal surface of human AEC'S
2. due to low transfection effeicent and inability of repeated administration hence retroviral vector is indicated
Retroviral vectors because of their ability to integrate into host cell genome to long expression and perhaps a cure and their fesability
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