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Examples Gene Therapy

In gene thera...

Question

In gene therapy for ADA deficiency why is a functional ADA "complementary" DNA introduced into lymphocytes?

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Solution

dear student,
first I will tell you the meaning of functional i.e. the cell that has ADA enzyme working effectively so in order to transfer it to the person whose cells are deficient of ADA enzyme. while genetically engineering lymphocyte this functional gene is introduced so give the capacity that it was lacking. since lymphocytes are mortal they are needed to be periodically infused.
regards

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Q. Which of the following is commonly used as a vector for introducing a DNA fragment in human lymphocytes during gene therapy for curing ADA deficiency?

Q. How many of the following statements are true?
I. An ADA deficient patient requires infusion of genetically engineered lymphocytes periodically.
II. There is no need for periodic infusion if the ADA gene is introduced at the early embryonic stage.
III. ADA gene is introduced into lymphocytes by using Ti plasmid.
IV. Gene therapy using retroviral vectors may cause cancers in some patients.
V. Bone marrow transplantation and enzyme replacement therapy are completely curative.

Q. Arrange the following steps of gene therapy for ADA deficiency in the correct sequence.
I) Infection of lymphocytes by genetically engineered retrovirus
II) Culture of lymphocytes
III) Isolation of lymphocytes from ADA deficient patient
IV) Genetically modified lymphocytes are injected back to patient

Q. Arrange the steps of ADA-deficiency treatment in sequence:
I. The lymphocytes with ADA cDNA is returned to the patient
II. The lymphocytes from the blood of the patient are grown in culture outside the body
III. A functional ADA cDNA (using retroviral vector) is introduced into the lymphocytes

What is the first step in gene therapy to treat ADA deficiency?

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Examples Gene Therapy

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