Research Progress toward Gene Therapy

By Miller, Julie Ann | Science News, August 24, 1985 | Go to article overview

Research Progress toward Gene Therapy


Miller, Julie Ann, Science News


Rapid advances in laboratory research during the last few months have made the rare immune system disorder called adenosine deaminase deficiency likely to be the target of the first U.S. experiments in human gene therapy. In as little as two months a group of researchers from several institutions, led by W. French Anderson of the National Institutes of Health (NIH) in Bethesda, Md., may be ready to propose an experiment in which a normal human gene for adenosine deaminase (ADA) is transferred into bone marrow cells, which will then be returned to a patient. Currently, persons with ADA deficiency die early in childhood unless they receive a bone marrow transplant from a suitable donor.

The most striking laboratory data so far demonstrate the "correction" of defective immune system cells taken from a youngster with ADA deficiency, R. Michael Blaese of NIH reported this week in Gmienden, Austria, at the Workshop on Primary Immunodeficiency Diseases. In the disease, the lack of ADA enzyme allows the buildup of 2' deoxyadenosine triphosphate, a chemical that is particularly detrimental to immune system cells, especially T cells. Blaese and Don Kohn have demonstrated that after the transfer of a normal ADA gene, T and B cells of the ADA patient act like normal immune system cells.

Although excited by the data, Anderson said in an interview, "We're not ready to treat this patient tomorrow. There are still a lot of things that need to be done." The researchers plan to make a formal proposal for a human gene-therapy experiment, he says, after they get results on monkey experiments, expected in the next two months.

Currently about a half dozen U.S. patients are candidates for such a genetransfer experiment. These are children for whom there is no suitable bone marrow donor. This situation contrasts with that for Lesch-Nyhan disease, another enzyme deficiency that had been considered a likely focus of early genetic engineering attempts. Progress there has been slowed by the recent failure of a bone marrow transplant from a normal donor to ameliorate the disease.

Another important advance toward gene therapy was reported by Anderson earlier this month in Los Angeles at a meeting on tissue-specific expression of cloned genes. He and NIH colleagues Philip Kantoff and Martin Eglitis transferred a gene into mouse bone marrow cells. When the cells were transplanted into mice whose own bone marrow had been destroyed, they repopulated the marrow and after four months continue to produce cells containing the foreign gene. Most important, the gene transplanted into the animal's B and T cells produces its characteristic protein. …

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

Items saved from this article

This article has been saved
Highlights (0)
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.”

Citations (0)
Some of your citations are legacy items.

Any citation created before July 30, 2012 will labeled as a “Cited page.” New citations will be saved as cited passages, pages or articles.

We also added the ability to view new citations from your projects or the book or article where you created them.

Notes (0)
Bookmarks (0)

You have no saved items from this article

Project items include:
  • Saved book/article
  • Highlights
  • Quotes/citations
  • Notes
  • Bookmarks
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, APA and Chicago citation styles.

(Einhorn, 1992, p. 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.

Cited article

Research Progress toward Gene Therapy
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

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, 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."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.

    Author Advanced search

    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.