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A long-sought goal in genetics has been to develop therapies that can use correctly functioning genes to replace genes with defects. If we had the technology to predictably modify our genomes, we would have the ability to cure many diseases instead of having to place people on medications for their entire lives.

For a long time, gene therapy has remained an elusive dream. But, in the past few years the dream has come closer to reality, especially in the case of ten children, who live because of researchers who kept that dream in sight (1).

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Figure 1. Random children

ResearchBlogging.org

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The genetically modified children that I’m writing about, suffer from a disease called Adenosine Deaminase (ADA) deficiency.  Adenosine deaminase normally acts in a biochemical pathway that breaks down nucleotides.  Defects in this gene can lead to the build up of toxic compounds that kill T cells, an important component of the immune system.  Without T cells,  children with ADA are unable to fight off disease, and consequently live very short, challenging lives.

ADA deficiency has long been a target for scientists interested in gene therapy for multiple reasons.  Helping these children is an important goal of course, but this disease has also been an attractive target because children with ADA can be treated through bone marrow transplants (2).  Bone marrow, with the right characteristics from a healthy donor, contains stem cells that can develop into T cells and provide an immune response.  We have learned how to genetically engineer bone marrow cells.  This made the idea of genetic modifications through bone marrow transplants, and using this method to treat children with ADA, an achievable goal. 

Still, it has taken several years since researchers first started working on this problem to see a clear, lasting, success.

In January, researchers published the results of a long-term study (1) demonstrating that gene therapy increased the immune functioning in nine of the ten children with ADA deficiency, who were treated in the study.  All ten children are alive in a median time of 4 years after treatment.  Most promising, in five of the children, the normal immune functions have been restored.

References

  1. A. Aiuti, F. Cattaneo, S. Galimberti, U. Benninghoff, B. Cassani, L. Callegaro, S. Scaramuzza, G. Andolfi, M. Mirolo, I. Brigida, A. Tabucchi, F. Carlucci, M. Eibl, M. Aker, S. Slavin, H. Al-Mousa, A. Al Ghonaium, A. Ferster, A. Duppenthaler, L. Notarangelo, U. Wintergerst, R. H. Buckley, M. Bregni, S. Marktel, M. G. Valsecchi, P. Rossi, F. Ciceri, R. Miniero, C. Bordignon, M.-G. Roncarolo (2009). Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency New England Journal of Medicine, 360 (5), 447-458 DOI: 10.1056/NEJMoa0805817
  2. D. B. Kohn, F. Candotti (2009). Gene Therapy Fulfilling Its Promise New England Journal of Medicine, 360 (5), 518-521 DOI: 10.1056/NEJMe0809614

Comments

  1. #1 Maria Von Lunton
    January 20, 2011

    I am one of the ten children this was done to.

  2. #2 gagan
    June 28, 2011

    but some them developed leukemia ……..

  3. #3 Sandra Porter
    June 29, 2011

    @gagan: That wouldn’t surprise me.

    Any idea how many?

  4. #4 TheFutureisToday
    July 18, 2011

    I love the thought of this as a whole, just imagine one day when we can engineer future scientists that will expand what we understand by multitudes I’m talking about engineered humans with IQ’s that nearly reach the 1000s! Just think how much humans can evolve and accomplish with 100’s of Einsteins and hundreds more that surpass that!

  5. #5 Nicholas
    Portland Oregon
    July 5, 2012

    By the time we have anything near multitudes of Einsteins, we will have a world full of hulking brutes engineered for war and conquest. Genetic engineering is to be nothing more than medical and cosmetic treatment on living organisms. That is if we are to survive as a species.

  6. #6 Olivia
    California
    December 23, 2012

    Nature knows what it’s doing. 100 Einsteins with IQ’s of 1,000, without the accompanying wisdom and compassion will be scary indeed. You can’t cheat nature and just jump forward without the humanity of the individual growing. There’s so much we don’t know. We know just enough to get ourselves into trouble, like we are doing with genetically modified food, causing tumor and cancer and more in lab animals.

  7. #7 Chrontius
    Sunny Florida
    February 11, 2013

    Nicholas, given the opportunity, I’d just as soon be one of those hulking brutes whose rippling muscles accent his artificially augmented intelligence. There’s no reason you can’t use designer biology to be brilliant *and* fit.

    Olivia: Nature knows how to make things work, but that method involves a *lot* of dying, and it should go without saying that I won’t see results in my natural lifetime. When talking about humans, I’m particularly uncomfortable with the idea of making individual sacrifices for a collective good.

    Why do you assume that anyone with an enhanced intellect will be lacking wisdom and compassion?

    Also, if you have found a study linking genetically modified food and cancer, by all means please share. I’ve heard plenty of rumors, but any proof is elusive.

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