2. Let me turn next to genetic therapies

And here, of course, there are two categories that have to be delineated. The one is what is called somatic cell and the other is germ line therapy. Somatic cell is treating the individual at the cellular level to correct a genetic deficiency. For example, if it becomes possible to do some genetic engineering in the near future, it may be possible to treat individuals who have diabetes not with insulin injections but by introducing the gene that expresses insulin, which they lack, into the pancreas. So that they will in fact be able to produce the insulin within their own bodies, without having an exogenous source of insulin either in injection or now in inhalation form.

And a lot of work is now being done at the somatic cell level with conditions like cystic fibrosis. The early results haven't been all that encouraging. The promise has not been fulfilled but the work proceeds patiently and sooner rather than later I think we will have effective genetic therapies for a limited number of conditions, especially those where there's a single enzyme that's missing or malfunctioning. That kind of somatic cell therapy raises few ethical concerns other than those having to do with safety and efficacy. Is it safe and does it work? And those are standard for any kind of new therapy, whether it's genetic or otherwise. We have always to be concerned about safety and efficacy.

But germ line therapy is very different, because when you get into the germ line, you make changes not in the individual alone but in that individual's progeny, not only for this generation but for generations to come. In other words, we begin to intervene directly in the evolutionary process. And this can happen in two ways, one negative and the other positive.

continued...


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