Which of the following statements are correct regarding gene therapy of ADA deficiency patient?
(i) Lymphocytes from the blood of the patient are grown in a culture outside the body, a functional ADA cDNA is then introduced into these lymphocytes by using a retroviral vector, which is subsequently returned to the patient.
(ii) Lymphocytes are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes.
(iii) If the gene isolate from bone marrow cells producing ADA is introduced into body cells at early embryonic stages, it could be a permanent cure.
(iv) If the gene isolate from bone marrow cells producing ADA from cells at early embryonic stages is introduced into patient bone marrow, it could be a permanent cure.
(v) If the gene isolate from bone marrow cells producing ADA is introduced into lymphocytes cells at early embryonic stages, it could be a permanent cure.
Text Solution
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Let’s check each statement:
(i) Correct — lymphocytes are cultured, ADA cDNA is inserted using retroviral vector, then returned
(ii) Correct — lymphocytes are not immortal, so repeated infusions are needed
(iii) Correct — introducing functional gene at early embryonic stage could give permanent cure (theoretical concept)
(iv) Incorrect — gene is not typically introduced into bone marrow cells at embryonic stage as stated
(v) Incorrect — lymphocytes are not used for permanent embryonic-stage cure concept
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