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Several techniques have been developed to treat blood disorders caused by genetic mutations.  Which of the following methods would be most effective in the treatment of a patient homozygous for a deleterious mutation in a gene coding for a subunit of hemoglobin?A.Replacement of the mutant gene with a functional copy in red blood cellsB.Treatment with a drug that increases red blood cell productionC.Introduction and activation of a functional hemoglobin gene in bone marrow stem cellsD.Single blood transfusion from a patient homozygous for the wild-type hemoglobin gene

Question

Several techniques have been developed to treat blood disorders caused by genetic mutations.  Which of the following methods would be most effective in the treatment of a patient homozygous for a deleterious mutation in a gene coding for a subunit of hemoglobin?A.Replacement of the mutant gene with a functional copy in red blood cellsB.Treatment with a drug that increases red blood cell productionC.Introduction and activation of a functional hemoglobin gene in bone marrow stem cellsD.Single blood transfusion from a patient homozygous for the wild-type hemoglobin gene

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Solution

The most effective method for treating a patient homozygous for a deleterious mutation in a gene coding for a subunit of hemoglobin would be C. Introduction and activation of a functional hemoglobin gene in bone marrow stem cells.

Here's why:

A. Replacement of the mutant gene with a functional copy in red blood cells - This method might not be effective because red blood cells do not have a nucleus and therefore do not contain DNA.

B. Treatment with a drug that increases red blood cell production - This method might not be effective because it does not address the underlying genetic issue. The new red blood cells would still contain the mutant gene.

C. Introduction and activation of a functional hemoglobin gene in bone marrow stem cells - This method would be most effective because it addresses the root cause of the problem. By introducing a functional gene into the bone marrow stem cells, which are responsible for producing new blood cells, this method ensures that all future red blood cells will contain the functional gene.

D. Single blood transfusion from a patient homozygous for the wild-type hemoglobin gene - This method might not be effective because it provides only a temporary solution. The patient's body would continue to produce red blood cells with the mutant gene.

This problem has been solved

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