Navigation Links
New gene therapy strategy boosts levels of deficient protein in Friedreich's ataxia
Date:7/25/2012

New Rochelle, NY, July 25, 2012A 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 Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zo 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 in the article "TALE Proteins Induce the Expression of the Frataxin Gene. (http://online.liebertpub.com/doi/full/10.1089/hum.2012.034)

"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.


'/>"/>
Contact: Vicki Cohn
vcohn@liebertpub.com
914-740-2100 x2156
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert  

Related biology news :

1. Stanford scientists develop gene therapy approach to grow blood vessels in ischemic limbs
2. Ottawa researchers to lead world-first clinical trial of stem cell therapy for septic shock
3. New insight into mechanisms behind autoimmune diseases suggests a potential therapy
4. The Japanese traditional therapy, honokiol, blocks key protein in inflammatory brain damage
5. Researchers discover novel therapy for Crohns disease
6. Nanotherapy: Treating deadly brain tumors by delivering big radiation with tiny tools
7. Cell therapy using patients own bone marrow may present option for heart disease
8. Harmless human virus may be able to boost the effects of chemotherapy
9. Minneapolis Heart Institute selected to participate in Cardiovascular Cell Therapy Research Network
10. Nanobubbles plus chemotherapy equals single-cell cancer targeting
11. New data suggests interferon-free therapy around the corner for HCV patients
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
New gene therapy strategy boosts levels of deficient protein in Friedreich's ataxia
(Date:2/1/2016)... 1, 2016  Today, the first day of American ... to develop a first of its kind workplace health ... Watson. In the first application of Watson ... IBM ), and Welltok will create a new offering ... cognitive analytics, delivered on Welltok,s health optimization platform. The ...
(Date:1/28/2016)... Jan. 28, 2016 Synaptics (NASDAQ: SYNA ), a ... its second quarter ended December 31, 2015. ... of fiscal 2016 increased 2 percent compared to the comparable quarter ... of fiscal 2016 was $35.0 million, or $0.93 per diluted share. ... income for the first quarter of fiscal 2016 grew 9 percent ...
(Date:1/25/2016)... Unisys Corporation (NYSE: UIS ) today announced the implementation ... New York City , to help U.S. ... the United States using passports that are ... of the system at Dulles last year. The system will ... 2016. --> pilot testing of the system at ...
Breaking Biology News(10 mins):
(Date:2/11/2016)...  Wellcentive today announced it has been selected ... -based community care organization (CCO) with more than ... reporting and care management solutions and services. Wellcentive,s ... quality managers, analysts and care managers while providing ... FamilyCare members. Oregon ...
(Date:2/11/2016)... , Feb. 11, 2016   BioInformant announces ... "Stem Cell Research Products, Opportunities, Tools, and Technologies – ... The ... the stem cell industry, BioInformant has more than a ... stem cell market, by stem cell type. This powerful ...
(Date:2/10/2016)... , Febr. 10, 2016 /PRNewswire/ - BioAmber Inc. (NYSE: ... to announce that Mitsui & Co. Ltd., its partner ... acid plant, is investing an additional CDN$25 million in ... its stake from 30% to 40%.  Mitsui will also ... acid produced in Sarnia , providing ...
(Date:2/10/2016)... 2016 ASAE is introducing a hybrid membership ... (AMC) the option of joining or renewing through an ... by staff size, every employee in any size association ... reap all available member benefits.   John ... membership options will allow organizations of any size and ...
Breaking Biology Technology: