Featured Research

from universities, journals, and other organizations

Novel Method Of Gene Replacement Reported By University Of Washington Researchers

Date:
April 1, 1998
Source:
University Of Washington
Summary:
Dr. David W. Russell, assistant professor of medicine, and Roli Hirata, research technician at the University of Washington, report the successful use of a modified virus to perform a novel method of gene replacement that may be an important step toward overcoming obstacles to efficient gene therapy.

Dr. David W. Russell, assistant professor of medicine, and Roli Hirata, research technician at the University of Washington, report the successful use of a modified virus to perform a novel method of gene replacement that may be an important step toward overcoming obstacles to efficient gene therapy. Their findings are reported in the April issue of Nature Genetics.

Related Articles


Until now, gene therapy researchers have focused on gene addition, using a variety of modified viruses as vectors or transport vehicles to "infect" and insert the proper genetic material into cell nuclei that have genes with undesirable mutations. While the proper genetic material is inserted, it goes to random locations on the chromosome, and the faulty genetic material also remains.

By contrast, Russell and Hirata were able to achieve efficient gene correction. They succeeded in targeting the exact location of the mutated gene on the chromosome and replacing it with the correct genetic material present in the viral vector, at exactly the right location.

Russell uses a typewriter analogy: the new method finds the typographical error, whites it out and types in the proper sequence in the right place.

"If you had a 'typo' in a financial statement, say a missing zero, you'd want to fix in the right place, not randomly anywhere in the statement," he explains. "Or if you were correcting an instruction booklet with an error in it, you'd want to insert the corrected instruction in the right place on the right page."

The UW researchers' method guarantees that the new gene is controlled by appropriate genetic circuitry, ensuring that it is switched on in the right cells, at the right time and at the right dosage.

In addition to targeting exactly the right part of the cell, the repaired copy and the mutated copy of the genetic materal were exchanged at what researchers consider a very high frequency, in approximately 1 percent of targeted cells. The highest rates occurred in normal human fibroblasts -- connective tissue cells.

"This level of exchange is perhaps 10,000 to 100,000 times better than has previously been achieved in normal human cells," said Russell. "There are some diseases such as hemophilia that could perhaps be cured with only modest improvements in the gene correction rate. Many diseases could be cured if future research enables us to correct genes in 10 percent of the targeted cells."

As a vector, the researchers used a virus called adeno-associated virus 2 (AAV), a single-stranded DNA virus capable of integrating into the chromosomes of mammals. Until this research, the potential of AAV vectors for gene targeting had not been explored. Russell and Hirata found that such vectors can target chromosomal genes at high frequencies and introduce modifications without creating additional mutations.

In an accompanying News & Views article, Dr. Alan Bernstein of the Samuel Lunenfeld Research Institute in Toronto states that the UW researchers' achievement is an important step toward efficient gene therapy, and that their results, in combination with similar advances on other fronts, suggest that the technical obstacles to efficient, targeted replacement of defective genes will eventually be overcome.

The research was supported by grants from the American Society of Hematology, the Lucile P. Markey Charitable Trust, the March of Dimes Birth Defects Foundation and the National Institutes of Health.


Story Source:

The above story is based on materials provided by University Of Washington. Note: Materials may be edited for content and length.


Cite This Page:

University Of Washington. "Novel Method Of Gene Replacement Reported By University Of Washington Researchers." ScienceDaily. ScienceDaily, 1 April 1998. <www.sciencedaily.com/releases/1998/04/980401074955.htm>.
University Of Washington. (1998, April 1). Novel Method Of Gene Replacement Reported By University Of Washington Researchers. ScienceDaily. Retrieved March 1, 2015 from www.sciencedaily.com/releases/1998/04/980401074955.htm
University Of Washington. "Novel Method Of Gene Replacement Reported By University Of Washington Researchers." ScienceDaily. www.sciencedaily.com/releases/1998/04/980401074955.htm (accessed March 1, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, March 1, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Rehab Robot Helps Restore Damaged Muscles and Nerves

Rehab Robot Helps Restore Damaged Muscles and Nerves

Reuters - Innovations Video Online (Mar. 1, 2015) A rehabilitation robot prototype to help restore deteriorated nerves and muscles using electromyography and computer games. Ben Gruber reports. Video provided by Reuters
Powered by NewsLook.com
How Facebook Use Can Lead To Depression

How Facebook Use Can Lead To Depression

Newsy (Mar. 1, 2015) Margaret Duffy of the University of Missouri talks about her study on the social network and the envy and depression that Facebook use can cause. Video provided by Newsy
Powered by NewsLook.com
Could a $34 Smartphone Device Improve HIV Diagnosis in Africa?

Could a $34 Smartphone Device Improve HIV Diagnosis in Africa?

Reuters - Innovations Video Online (Feb. 27, 2015) A dongle that plugs into a Smartphone mimics a lab-based blood test for HIV and syphilis and can detect the diseases in 15 minutes, say researchers. Tara Cleary reports. Video provided by Reuters
Powered by NewsLook.com
Doctor Says Head Transplants Possible Within Two Years

Doctor Says Head Transplants Possible Within Two Years

Buzz60 (Feb. 27, 2015) An Italian doctor is saying he could stick someone&apos;s head onto someone else&apos;s body. Patrick Jones (@Patrick_E_Jones) reports. Video provided by Buzz60
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins