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Chapter 14. Genetic Therapeutics

A child is born with a cleft lip and palate, a congenital heart malformation, and a missing radius. The parents ask you (the attending physician) if gene therapy is an option available for their child. You would answer:

A. You can refer them now for gene therapy to correct the defects.

B. Gene therapy is not quite available, but should be available in a couple of years to fix the problems.

C. Gene therapy is not currently available, but even if it were, it would be unlikely to correct congenital malformations.

D. Gene therapy is available but cost prohibitive.

E. Gene therapy is ethically wrong, and you would discourage them from even considering it.

C is the correct answer.

  • A. Gene therapy is currently not a standardly available mode of therapy.

  • B. Congenital malformations are not likely to be helped by gene therapy–this would only work if the defects were known ahead of time and the genetic manipulation performed at or around the time of conception—in the embryo.

  • D. Gene therapy is not standardly available regardless of the issue of being cost prohibitive.

  • E. Of course patients should not be counseled in such a directive manner.

You are asked to speak to a civic group in your home community. They want information about "cloning." What would be good information to share with them?

A. All forms of cloning are morally repugnant and should be outlawed.

B. Everyone should have a clone of themselves made and preserved in case organ donation is needed in the future.

C. The exact cloning of an individual is something that will not happen as we are more than just our genes.

D. The cloning of Dolly the sheep was accomplished with no unexpected complications.

E. Cloning should not be considered a controversial issue–all major religious and legal organizations have endorsed it.

C is the correct answer.

  • A. Cloning can be a non-controversial mode of therapy.

  • B. Even if this technology were available, the ethical issues are yet to be resolved.

  • D. The cloning of Dolly the sheep was accomplished, but there was the unexpected complication of early aging.

  • E. Cloning is an extremely controversial issue.

You see a 13-year-old boy in your clinic. He is deeply upset over his size. After you assess the family you note that he has familial short stature, i.e., ...

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