Consider the following statements:

1. Genetic medicines can use engineered viruses as carriers.

2. Genetic medicine typically replaces a patients entire DNA sequence.

3. Ex vivo gene therapy modifies cells outside the body before returning them to the patient.

Which of the statements given above is/are correct?A.1 and 3 onlyB.1 and 2 onlyC.2 and 3 onlyD.1, 2 and 3

Question

Consider the following statements: 1. Genetic medicines can use engineered viruses as carriers. 2. Genetic medicine typically replaces a patients entire DNA sequence. 3. Ex vivo gene therapy modifies cells outside the body before returning them to the patient. Which of the statements given above is/are correct?

Accepted Answer

1 and 3 only. Statements 1 and 3 are correct. MedlinePlus says viruses modified so they can't cause disease are used as vectors, so statement 1 holds. Bulcha and colleagues describe ex vivo therapy as taking out a patient's cells, changing them with a vector and infusing them back. MedlinePlus describes the same steps, so statement 3 holds. MedlinePlus says genome editing alters genetic material at precise locations in the genome. So statement 2, about replacing a patient's entire DNA sequence, is wrong.

How is the gene therapy Casgevy made for each patient, according to the UK regulator and the US FDA?

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