Featured Research

from universities, journals, and other organizations

New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia

Date:
July 25, 2012
Source:
Mary Ann Liebert, Inc., Publishers
Summary:
A novel approach to gene therapy that instructs a person’s own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich’s ataxia.

A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published in Human Gene Therapy.

Related Articles


The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zoé Coulombe, and Joel Rousseau, Laval University, Quebec,and University of Quebec, Canada, takes advantage of the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. As a model of how this method could be used to treat genetic disease, the authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich's ataxia.  The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, conclude the authors.

"This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.


Story Source:

The above story is based on materials provided by Mary Ann Liebert, Inc., Publishers. Note: Materials may be edited for content and length.


Journal Reference:

  1. Jacques P. Tremblay, Pierre Chapdelaine, Zoé Coulombe, Joel Rousseau. Transcription Activator-Like Effector Proteins Induce the Expression of the Frataxin Gene. Human Gene Therapy, 2012; 120720084321004 DOI: 10.1089/hum.2012.034

Cite This Page:

Mary Ann Liebert, Inc., Publishers. "New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia." ScienceDaily. ScienceDaily, 25 July 2012. <www.sciencedaily.com/releases/2012/07/120725105212.htm>.
Mary Ann Liebert, Inc., Publishers. (2012, July 25). New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia. ScienceDaily. Retrieved March 27, 2015 from www.sciencedaily.com/releases/2012/07/120725105212.htm
Mary Ann Liebert, Inc., Publishers. "New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia." ScienceDaily. www.sciencedaily.com/releases/2012/07/120725105212.htm (accessed March 27, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Friday, March 27, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

House Ready to Pass Medicare Doc Bill

House Ready to Pass Medicare Doc Bill

AP (Mar. 26, 2015) — In rare bipartisan harmony, congressional leaders pushed a $214 billion bill permanently blocking physician Medicare cuts toward House passage Thursday, moving lawmakers closer to resolving a problem that has plagued them for years. (March 26) Video provided by AP
Powered by NewsLook.com
HIV Outbreak Prompts Public Health Emergency In Indiana

HIV Outbreak Prompts Public Health Emergency In Indiana

Newsy (Mar. 26, 2015) — Indiana Gov. Mike Pence says he will bring additional state resources to help stop the epidemic. Video provided by Newsy
Powered by NewsLook.com
Indiana Permits Needle Exchange as HIV Cases Skyrocket

Indiana Permits Needle Exchange as HIV Cases Skyrocket

Reuters - US Online Video (Mar. 26, 2015) — Governor Mike Pence declares the recent HIV outbreak in rural Indiana a "public health emergency" and authorizes a short-term needle-exchange program. Rough Cut (no reporter narration) Video provided by Reuters
Powered by NewsLook.com
AAA: Distracted Driving a Serious Teen Problem

AAA: Distracted Driving a Serious Teen Problem

AP (Mar. 25, 2015) — While distracted driving is not a new problem for teens, new research from the AAA Foundation for Traffic Safety says it&apos;s much more serious than previously thought. (March 25) 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