Featured Research

from universities, journals, and other organizations

Jefferson Scientists Hope To Perfect Gene Therapy Without Viruses

Date:
August 28, 2000
Source:
Jefferson Medical College
Summary:
Gene therapy – which aims to replace "bad" genes with useful ones – has yet to live up to its promise largely because of problems delivering genes to the right place in the body. What’s more, the viruses many gene therapy techniques use can arouse unwanted immune reactions. Researchers at Jefferson Medical College have developed a system that sidesteps viruses, and they hope, some of the inherent problems with their use.

Gene therapy – which aims to replace "bad" genes with useful ones – has yet to live up to its promise largely because of problems delivering genes to the right place in the body. What’s more, the viruses many gene therapy techniques use can arouse unwanted immune reactions.

Researchers at Jefferson Medical College have developed a system that sidesteps viruses, and they hope, some of the inherent problems with their use. Scientists, led by Eric Wickstrom, PhD, professor of microbiology and immunology, and graduate research assistant Stephen H. Cleaver, both of Jefferson Medical College and Thomas Jefferson University in Philadelphia, have used DNA and bacterial proteins to deliver a gene to a specific place in a piece of human DNA.

They have used a "transposon," or a naturally mobile piece of DNA, from the E. coli bacterium as a gene delivery vehicle, inserting a gene for antibiotic resistance into a segment of DNA.

"This is the first time it [gene therapy] was done this way," says Dr. Wickstrom, who is also a member of Jefferson’s Kimmel Cancer Center. Their results appear August 22 in the journal Gene.

"The transposon inserted in exactly the right place and expressed its product, in this case, an antibiotic-resistant gene." The technique has several advantages, he says. "It avoids using viruses that cause immune reactions, it puts the gene in a precise, known location as opposed to anywhere in the cell’s DNA, and it makes only one copy."

There are significant problems with viral methods, Dr. Wickstrom says. In traditional gene therapy methods, there is a question of how many gene copies may be made in each individual and where in the cell’s DNA a copy may go. "The genes could go anywhere in the genome. This may be okay, or they could interact inappropriately with other genes, such as a tumor suppressor gene, knocking out its function and starting a cancer."

Dr. Wickstrom points out that his current tests so far have been only in bacteria to date. "We would like to avoid these problems and still have an effective method," he explains. "We’ve only done this in bacteria – not a higher organism, though this was with a human genomic sequence."

The Jefferson team was awarded a patent for the technique and a new grant for more than $660,000 from the National Institutes of Health to continue to develop their method of gene transfer in yeast and mice.

Dr. Wickstrom notes that gene therapy with and without viruses currently is being studied in clinical trials by many researchers in this country and overseas. In his recent book, "Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors," he and his co-authors described a variety of preclinical and clinical studies in the United States and other countries.

He adds "the ability to turn off or correct individual disease-causing genes, or to replace them at will in a patient’s cells provides a powerful therapeutic intervention in genetic diseases.

"It’s clear that all disease results from incorrect gene expression, one way or another," he notes. "By turning off some genes or correcting others, we may be able to treat each disease at its point of origin." Eventually, Dr. Wickstrom hopes to use his nonviral method to provide normally functioning genes in diseases such as hemophilia, sickle cell anemia, muscular dystrophy, and phenylketonuria, and therapeutic genes to treat cancer.


Story Source:

The above story is based on materials provided by Jefferson Medical College. Note: Materials may be edited for content and length.


Cite This Page:

Jefferson Medical College. "Jefferson Scientists Hope To Perfect Gene Therapy Without Viruses." ScienceDaily. ScienceDaily, 28 August 2000. <www.sciencedaily.com/releases/2000/08/000823082148.htm>.
Jefferson Medical College. (2000, August 28). Jefferson Scientists Hope To Perfect Gene Therapy Without Viruses. ScienceDaily. Retrieved September 30, 2014 from www.sciencedaily.com/releases/2000/08/000823082148.htm
Jefferson Medical College. "Jefferson Scientists Hope To Perfect Gene Therapy Without Viruses." ScienceDaily. www.sciencedaily.com/releases/2000/08/000823082148.htm (accessed September 30, 2014).

Share This



More Health & Medicine News

Tuesday, September 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

How 'Yes Means Yes' Defines Sexual Assault

How 'Yes Means Yes' Defines Sexual Assault

Newsy (Sep. 29, 2014) Aimed at reducing sexual assaults on college campuses, California has adopted a new law changing the standard of consent for sexual activity. Video provided by Newsy
Powered by NewsLook.com
Scientists May Have Found An Early Sign Of Pancreatic Cancer

Scientists May Have Found An Early Sign Of Pancreatic Cancer

Newsy (Sep. 29, 2014) Researchers looked at 1,500 blood samples and determined people who developed pancreatic cancer had more branched chain amino acids. Video provided by Newsy
Powered by NewsLook.com
Colo. Doctors See Cluster of Enterovirus Cases

Colo. Doctors See Cluster of Enterovirus Cases

AP (Sep. 29, 2014) Doctors at the Children's Hospital of Colorado say they have treated over 4,000 children with serious respiratory illnesses since August. Nine of the patients have shown distinct neurological symptoms, including limb weakness. (Sept. 29) Video provided by AP
Powered by NewsLook.com
Dr.'s Unsure of Cause of Fast-Spreading Virus

Dr.'s Unsure of Cause of Fast-Spreading Virus

AP (Sep. 29, 2014) Doctors at the Children's Hospital of Colorado say they have treated over 4,000 children with serious respiratory illnesses since August. Nine of the patients have shown distinct neurological symptoms, including limb weakness. (Sept. 29) Video provided by AP
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