Gene Therapy Not Needed

Gene Therapy Not Needed

by Dr. Hsien-Hsien Lei
Posted June 3, 2007 in DNA and Disease, Gene Therapy

The genome revolution isn’t all about re-engineering our genome. In a mis-titled article–Gene therapy: how science moved from hype to hope–Mark Henderson of The Times UK talks about the many different gene variants that have been identified recently that are common in the general population and yield a smaller effect compared to major gene mutations affecting only a small subset of the population, e.g., Huntington’s or cystic fibrosis. But rather than focusing on actually changing our genes, as the title would suggest, the article concludes with the idea that environmental factors are easier to manipulate than genes because gene variants with smaller effects are neither sufficient nor necessary for causing disease. I’m glad this point was emphasized so that people don’t fall into the trap of believing in genetic determinism.

As for the commenter who wondered why research focuses on disease-causing genes rather than disease-preventing, there’s an easy answer. Eliminating disease is what the medical model is based on. Perhaps the genome revolution will bring about another revolution in preventive medicine.
clipped from

Where genetics was once capable of pinning down only rare mutations with a catastrophic impact, it is now tracing variants with smaller effects that are much more widespread. You might call it the democratisation of the genome.

“What we are going to get out of this in the end is not genetic engineering, but environmental engineering,” Dr Plomin said. “A lot is going to be about changing behaviour, about education. It’s not always going to be: you’ve got this genetic problem and here’s a pill. Most genetic effects are going to be too small for that.”

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Comment by Steven Murphy MD Subscribed to comments via email

Dr Eric Topol of Scripps wrote an article a few months ago on GWAS regarding “healthy” genes. Nirs Barzilai at Albert Einstein has focused on these same things “Centenarian studies” What he found was these people who lived longer than 100 were in fact smokers and drinkers. Their genes helped them defend against these insults. Predictive, Preventative, Personalized Medicine is where we are all headed.

Comment by Hsien

“What he found was these people who lived longer than 100 were in fact smokers and drinkers.”

Ahhh. The irony of life.

Comment by Andrew Sun

Genetic Determinism…there is genetic determinism in every piece of sci-fi work. People just like genetic determinism (or hate), but they don’t always think of it, unfortunately

Comment by Hsien

Hi Andrew! I would personally like to blame my genes for everything that’s gone wrong in my life. On the other hand, I can’t really claim that things have gone badly wrong either. ;)


[...] Genie, a blog carnival on genetics, genes, DNA, and the genome over at Eye on DNA. clipped from Infants and Autism and Education at Autism Vox Kristina Chew looks at diagnosis for autism [...]

Comment by Roger Anderson Subscribed to comments via email

This is a topic I have not followed closely for some time. I have been away from the bench for over a decade. I think that a very important but difficult to confront issue is that of the introduction of new gene variants into the population. I am all for curing a disease but not for introducing new alleles. That means that anyone who receives gene-therapy must not reproduce. How will that be enforced – ouch – sterilization. Otherwise the risk to the general population is too great.

Just my thoughts on the subject.

Comment by Hsien

Hi and welcome, Roger! I’ve also been away from the bench since 1999. Can’t believe time has passed so quickly.

The thing is, new gene variants ARE being introduced all the time by nature. In fact, I wonder if we are better able to control the creation of good mutations vs bad although there’s really no way any of us can know for certain. And as for gene therapy, most–if not all–will target somatic cells instead of germ cells so we should be safe there.

Comment by Roger Anderson Subscribed to comments via email

Yes, nature contributes a change every 10,000 bases or so. The difference is that no one is legally responsible for that. When we begin introducing intentional changes we become liable for them. Yet we have little control over them once they are unleashed.

Targeting is a hope not yet a reality as far as I understand. Perhaps if the change is introduced in vitro and then the cells reintroduced to the host we would be safer.

(Comments wont nest below this level)

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