Research Progress toward Gene Therapy

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

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

Sign up now for a free, 1-day trial and receive full access to:

  • Questia's entire collection
  • Automatic bibliography creation
  • More helpful research tools like notes, citations, and highlights
  • Ad-free environment

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.
Loading One moment ...
Project items
Notes
Cite this article

Cited article

Style
Citations are available only to our active members.
Sign up now to cite pages or passages in MLA, APA and Chicago citation styles.

Cited article

Research Progress toward Gene Therapy
Settings

Settings

Typeface
Text size Smaller Larger
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?

While we understand printed pages are helpful to our users, this limitation is necessary to help protect our publishers' copyrighted material and prevent its unlawful distribution. We are sorry for any inconvenience.
Full screen

matching results for page

Cited passage

Style
Citations are available only to our active members.
Sign up now to cite pages or passages in MLA, APA and Chicago citation styles.

Cited passage

Welcome to the new Questia Reader

The Questia Reader has been updated to provide you with an even better online reading experience.  It is now 100% Responsive, which means you can read our books and articles on any sized device you wish.  All of your favorite tools like notes, highlights, and citations are still here, but the way you select text has been updated to be easier to use, especially on touchscreen devices.  Here's how:

1. Click or tap the first word you want to select.
2. Click or tap the last word you want to select.

OK, got it!

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.

For full access in an ad-free environment, sign up now for a FREE, 1-day trial.

Already a member? Log in now.

Are you sure you want to delete this highlight?