Cystic Fibrosis Controversy: A New Theory Hints That Gene Therapy in the Womb Can Cure Disease

By Travis, John | Science News, May 10, 1997 | Go to article overview

Cystic Fibrosis Controversy: A New Theory Hints That Gene Therapy in the Womb Can Cure Disease


Travis, John, Science News


"Cystic fibrosis is a preventable disease."

J. Craig Cohen makes the statement casually, almost as if unaware of the startling and controversial implications of his words. Yet Cohen certainly realizes that he has challenged the conventional wisdom about what causes this fatal disease and how to treat it.

"There's a large body of people out there who, if we're right, are pretty wrong," says Cohen, a scientist at Louisiana State University Medical Center in New Orleans.

In the March 1 Lancet, Cohen, Janet E. Larson of the Alton Ochsner Medical Foundation in New Orleans, and their colleagues reported that they had cured mice that have a mutant gene similar to the one that causes cystic fibrosis in people.

Surprisingly, the researchers did not permanently replace the defective gene in their mice. Instead, they temporarily added a working version of the gene to developing mouse fetuses.

About a week before the birth of each mouse, the researchers, using a novel form of gene therapy somewhat like amniocentesis, pierced the mother's amniotic sac and injected into the amniotic fluid genetically engineered viruses harboring the therapeutic gene. By breathing in this fluid, the fetuses exposed their lungs and gut to the gene, which then presumably directed cells there to produce its protein, says Cohen.

The viruses used do not integrate their genetic material into host cells, note the researchers. In fact, tests showed that the activity of the therapeutic gene in cells lasted only a few days. Yet even this brief presence, say Cohen and Larson, was enough to cure 13 out of 13 otherwise doomed mice.

The researchers conclude from this gene therapy study that cystic fibrosis is a disease in which the lungs and other organs develop improperly because of mutations in the cystic fibrosis gene, cftr. In essence, cystic fibrosis is a birth defect that leads to death years later.

Cohen and Larson believe that a normal cftr is needed during embryogenesis for the development of secretory cells that they have recently identified. Without these cells, the symptoms of cystic fibrosis develop, they contend.

This viewpoint differs sharply from the detailed picture of the disease that has emerged from other research within the last year (SN: 5/4/96, p. 279).

While cystic fibrosis can damage many organs, its hallmark in people is lung problems. Most scientists believe that mutant versions of cftr encode defective proteins that cannot properly transport chloride ions into lung cells, thus creating a buildup of salt outside them. This abnormally salty environment disables a natural antibiotic, leading to bacterial infections that trigger the production of mucus in the airways. Ultimately, lung damage from the infections and the accumulation of mucus make breathing impossible.

This perspective on cystic fibrosis has spurred gene therapists to focus on fixing lung cells by replacing mutant cftr genes with functional versions. In theory, that should reduce salt buildup and eliminate the deadly symptoms of the disease. Yet the first clinical trials of this gene therapy have not met with much success (SN: 10/28/95, p. …

The rest of this article is only available to active members of Questia

Already a member? Log in now.

Notes for this article

Add a new note
If you are trying to select text to create highlights or citations, remember that you must now click or tap on the first word, and then click or tap on the last word.
One moment ...
Default project is now your active project.
Project items
Notes
Cite this article

Cited article

Style
Citations are available only to our active members.
Buy instant access to cite pages or passages in MLA 8, MLA 7, APA and Chicago citation styles.

(Einhorn, 1992, p. 25)

(Einhorn 25)

(Einhorn 25)

1. Lois J. Einhorn, Abraham Lincoln, the Orator: Penetrating the Lincoln Legend (Westport, CT: Greenwood Press, 1992), 25, http://www.questia.com/read/27419298.

Note: primary sources have slightly different requirements for citation. Please see these guidelines for more information.

Cited article

Cystic Fibrosis Controversy: A New Theory Hints That Gene Therapy in the Womb Can Cure Disease
Settings

Settings

Typeface
Text size Smaller Larger Reset View mode
Search within

Search within this article

Look up

Look up a word

  • Dictionary
  • Thesaurus
Please submit a word or phrase above.
Print this page

Print this page

Why can't I print more than one page at a time?

Help
Full screen
Items saved from this article
  • Highlights & Notes
  • Citations
Some of your highlights are legacy items.

Highlights saved before July 30, 2012 will not be displayed on their respective source pages.

You can easily re-create the highlights by opening the book page or article, selecting the text, and clicking “Highlight.”

matching results for page

    Questia reader help

    How to highlight and cite specific passages

    1. Click or tap the first word you want to select.
    2. Click or tap the last word you want to select, and you’ll see everything in between get selected.
    3. You’ll then get a menu of options like creating a highlight or a citation from that passage of text.

    OK, got it!

    Cited passage

    Style
    Citations are available only to our active members.
    Buy instant access to cite pages or passages in MLA 8, MLA 7, APA and Chicago citation styles.

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences." (Einhorn, 1992, p. 25).

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences." (Einhorn 25)

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences." (Einhorn 25)

    "Portraying himself as an honest, ordinary person helped Lincoln identify with his audiences."1

    1. Lois J. Einhorn, Abraham Lincoln, the Orator: Penetrating the Lincoln Legend (Westport, CT: Greenwood Press, 1992), 25, http://www.questia.com/read/27419298.

    Cited passage

    Thanks for trying Questia!

    Please continue trying out our research tools, but please note, full functionality is available only to our active members.

    Your work will be lost once you leave this Web page.

    Buy instant access to save your work.

    Already a member? Log in now.

    Search by... Author
    Show... All Results Primary Sources Peer-reviewed

    Oops!

    An unknown error has occurred. Please click the button below to reload the page. If the problem persists, please try again in a little while.