Featured Research

from universities, journals, and other organizations

University Of Iowa Researchers Find Potential Way To Improve Gene Therapy Delivery To Brain

Date:
March 22, 2000
Source:
University Of Iowa
Summary:
Developing successful gene therapy for neurodegenerative diseases that affect a large proportion of the brain is challenging. One hurdle involves ensuring that the gene vectors -- disabled viruses that carry the genes -- are well distributed in the brain and efficiently deposit the genes in the target cells. However, University of Iowa and National Institutes of Health (NIH) investigators have collaborated to find that a certain gene vector can effectively reach many brain sites following a single injection.

IOWA CITY, Iowa -- Developing successful gene therapy for neurodegenerative diseases that affect a large proportion of the brain is challenging. One hurdle involves ensuring that the gene vectors -- disabled viruses that carry the genes -- are well distributed in the brain and efficiently deposit the genes in the target cells. However, University of Iowa and National Institutes of Health (NIH) investigators have collaborated to find that a certain gene vector can effectively reach many brain sites following a single injection.

Related Articles


The finding, based on animal models, recently appeared online at the website of the Proceedings of the National Academy of Sciences (PNAS). The print article will appear in the March 28 issue of the Proceedings.

"We did not believe that multiple injections of the gene therapy vector was the answer to achieving widespread distribution throughout the brain," said Beverly Davidson, Ph.D., UI professor of internal medicine and the study's lead investigator. "From previous work using tissue culture, we believed that different types of adeno-associated virus (AAV) vectors bound to different receptors and could therefore behave differently when placed into brain tissues."

Subtle differences in the protein coats of the AAV-based vectors give them distinct properties, Davidson said.

"In the present study, we found surprising characteristics in AAV5, one of the AAV-based vectors tested," she said. "The AAV5 had a unique ability to spread very far beyond the injection site after being introduced into the brain."

Davidson said the data are exciting because they suggest that AAV5-based vectors could be used to deliver correct copies of genes to cells throughout the central nervous system without the need for multiple injections.

The next step is to test the AAV5 vector in animal models of neurodegenerative diseases. For these studies, DNA sequences encoding therapeutic molecules will be placed into the AAV5 vectors.

The AAV5 type might eventually have particular application for gene therapy treatment of disorders where a large proportion of the brain is affected, such as Alzheimer's disease, Huntington's disease and lysosomal storage diseases such as Batten disease (a neurodegenerative disease with childhood onset).

In addition to Davidson, Colleen S. Stein, Ph.D., postdoctoral fellow in Davidon's laboratory, was also a major contributor to the work. John A. Chiorini, Ph.D., a researcher in the Gene Therapy and Therapeutics Branch of the National Institute of Dental and Craniofacial Research at the NIH, also collaborated on the study.

The study was supported in part by an NIH grant. Davidson is also a fellow of the Roy J. Carver Trust.

University of Iowa Health Care describes the partnership between the UI College of Medicine and the UI Hospitals and Clinics and the patient care, medical education and research programs and services they provide.

NOTE TO EDITORS: Copies of the journal article can be downloaded from the PNAS website at http://www.pnas.org/cgi/content/full/050581197


Story Source:

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


Cite This Page:

University Of Iowa. "University Of Iowa Researchers Find Potential Way To Improve Gene Therapy Delivery To Brain." ScienceDaily. ScienceDaily, 22 March 2000. <www.sciencedaily.com/releases/2000/03/000322091215.htm>.
University Of Iowa. (2000, March 22). University Of Iowa Researchers Find Potential Way To Improve Gene Therapy Delivery To Brain. ScienceDaily. Retrieved December 18, 2014 from www.sciencedaily.com/releases/2000/03/000322091215.htm
University Of Iowa. "University Of Iowa Researchers Find Potential Way To Improve Gene Therapy Delivery To Brain." ScienceDaily. www.sciencedaily.com/releases/2000/03/000322091215.htm (accessed December 18, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Thursday, December 18, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Kids Die While Under Protective Services

Kids Die While Under Protective Services

AP (Dec. 18, 2014) As part of a six-month investigation of child maltreatment deaths, the AP found that hundreds of deaths from horrific abuse and neglect could have been prevented. AP's Haven Daley reports. (Dec. 18) Video provided by AP
Powered by NewsLook.com
UN: Up to One Million Facing Hunger in Ebola-Hit Countries

UN: Up to One Million Facing Hunger in Ebola-Hit Countries

AFP (Dec. 17, 2014) Border closures, quarantines and crop losses in West African nations battling the Ebola virus could lead to as many as one million people going hungry, UN food agencies said on Wednesday. Duration: 00:52 Video provided by AFP
Powered by NewsLook.com
When You Lose Weight, This Is Where The Fat Goes

When You Lose Weight, This Is Where The Fat Goes

Newsy (Dec. 17, 2014) Can fat disappear into thin air? New research finds that during weight loss, over 80 percent of a person's fat molecules escape through the lungs. Video provided by Newsy
Powered by NewsLook.com
Why Your Boss Should Let You Sleep In

Why Your Boss Should Let You Sleep In

Newsy (Dec. 17, 2014) According to research out of the University of Pennsylvania, waking up for work is the biggest factor that causes Americans to lose sleep. Video provided by Newsy
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