Navigation Links
Friedreich's ataxia -- an effective gene therapy in an animal model
Date:4/6/2014

The transfer, via a viral vector, of a normal copy of the gene deficient in patients, allowed to fully and very rapidly cure the heart disease in mice. These findings are published in Nature Medicine on 6 April, 2014.

Friedreich's ataxia is a severe, rare hereditary disorder which combines progressive neuro-degeneration, impaired heart function and an increased risk of diabetes. The condition affects one in every 50,000 birth. There is currently no effective treatment for this disease. In most cases, Friedreich's ataxia starts in adolescence with impaired balance and coordination (ataxia) of voluntary arm and leg movements, confining the majority of patients to a wheelchair after 10-20 years' progression. However, complications affecting the heart are the major cause of death in 60% of patients, most often before the 35 years of age.

The disease is caused by a common mutation in the FXN gene which leads to a dramatic decrease in the production of the protein named 'frataxin'. The reduced frataxin level disturbs the activity of mitochondria. These organelles are essential to cells and play a fundamental role in energy production. The nerve tissue (cerebellum, spinal cord etc.) and heart tissue are particularly vulnerable to this shortage of energy, which can lead to fatal heart failure.

The teams led by Hlne Puccio, director of research at Inserm and Patrick Aubourg have developed a therapeutic approach based on the use of an adeno-associated virus (AAV)[1], which is known to efficiently target and express a therapeutic gene in heart cells. The virus was modified to make it harmless but nevertheless capable of introducing a normal copy of the FXN gene in the heart cells, thus leading to the expression of frataxin.

Hlne Puccio's team tested the efficacy of this treatment in a mouse model that faithfully reproduces the heart symptoms of patients suffering from Friedreich's ataxia. The results show that a single int
'/>"/>

Contact: Hélène Puccio
hpuccio@igbmc.fr
33-388-653-264
INSERM (Institut national de la sant et de la recherche mdicale)
Source:Eurekalert  

Page: 1 2

Related biology news :

1. New gene therapy strategy boosts levels of deficient protein in Friedreichs ataxia
2. Interferon-beta aids balance and movement in mice with spinocerebellar ataxia 7
3. Patients and scientists join forces to tackle Friedreichs Ataxia
4. International patient alliance to fund Spanish Friedreichs ataxia gene-therapy project
5. More effective method of imaging proteins
6. New discovery may lead to effective prevention and treatment of graft-versus-host dsease
7. Urgent research needed to determine most effective follow-up care for lung cancer patients
8. As deadly cat disease spreads nationally, MU veterinarian finds effective treatment
9. Lab tests show arthritis drug effective against global parasite
10. Novel discovery by NUS scientists paves the way for more effective treatment of cancers
11. An economical, effective and biocompatible gene therapy strategy promotes cardiac repair
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Friedreich's ataxia -- an effective gene therapy in an animal model
(Date:10/29/2014)... Online registration is open for the ... Scientific Meeting. SIR 2015 is the one ... trainees and related health care professionals can ... image-guided science and technologies being presented, discussed ... community in minimally invasive, image-guided medicine, SIR ...
(Date:10/29/2014)... household and industrial products are in the environment – ... of them, scientists have yet to determine whether they ... toward doing that by estimating which substances people are ... in the ACS journal Environmental Science & Technology ... the risks to human health of any given substance ...
(Date:10/29/2014)... LANSING, Mich. – As bodies decompose, their types ... the clues they provide could mean the difference ... , Michigan State University is using a more ... help Detroit death-scene investigators examine these changing populations. ... as geographical location of death, gender, race, socioeconomic ...
Breaking Biology News(10 mins):SIR 2015: Interventional radiology shapes tomorrow's medicine 2MSU partners with Detroit to investigate death scenes 2
... FL, January 13, 2014 Scientists from the Florida campus ... of drug candidates that advance the search for new treatments ... drug scaffolds, described in a recent edition of The ... act on a demonstrated therapeutic target, the kappa opioid receptor ...
... have devised a way to measure the mass of ... one millionth of a trillionth of a gram. Weighing ... components of cells, could help researchers better understand their ... technology previously developed by Scott Manalis, an MIT professor ...
... say they practice some form of yoga and/or meditation. ... these practices seems unknown or elusive to many practitioners ... during meditation practice , by Fred Travis, PhD, Director ... at Maharishi University of Management, provides an overview ...
Cached Biology News:Scientists develop promising drug candidates for pain, addiction 2Weighing particles at the attogram scale 2Weighing particles at the attogram scale 3Weighing particles at the attogram scale 4Transcendental experiences during meditation 2Transcendental experiences during meditation 3
(Date:10/26/2014)... (PRWEB) October 26, 2014 The report ... by Application - Global Trends & Forecast to 2019", ... analysis and forecasting of volume consumed and value generated. ... 4,253.9 KT in 2014 to 6,701.3 KT by 2019, ... , Browse 91 market data tables and 22 figures ...
(Date:10/25/2014)... Los Angeles, California (PRWEB) October 23, 2014 ... VDRM ), a specialty pharmaceutical company devoted to ... pursuing out-licensing partnerships in 2015. , ViaDerma has ... method, which allows for rapid mass transfer of ... into the body to provide immediate localized therapy. ...
(Date:10/25/2014)... October 24, 2014 According to ... by Data Center Types (Colocation, Enterprise, Telecom), by ... (Tier 1, Tier 2, Tier 3, Tier 4) ... defines and segments the Data Center Construction Market ... forecasting of revenues. This research report also identifies ...
(Date:10/25/2014)... Va. (PRWEB) October 24, 2014 ... the nation’s leading nonprofit authority on the ... healthcare information exchange, announces its appointment of four ... Workgroup. The workgroup leaders include:, , ... ,     Erik Pupo, Specialist Leader, Deloitte Consulting ...
Breaking Biology Technology:Protective Coatings Market Poised to Reach $18,431.8 Million by 2019 - Report by MarketsandMarkets 2Protective Coatings Market Poised to Reach $18,431.8 Million by 2019 - Report by MarketsandMarkets 3Protective Coatings Market Poised to Reach $18,431.8 Million by 2019 - Report by MarketsandMarkets 4ViaDerma, Inc. Announces Plans to Pursue Out-Licensing Partnerships in 2015 2ViaDerma, Inc. Announces Plans to Pursue Out-Licensing Partnerships in 2015 3Data Center Construction Market Worth $22.73 Billion by 2019 - New Report by MarketsandMarkets 2Data Center Construction Market Worth $22.73 Billion by 2019 - New Report by MarketsandMarkets 3Data Center Construction Market Worth $22.73 Billion by 2019 - New Report by MarketsandMarkets 4WEDI Appoints Four New Security and Privacy Workgroup Co-chairs 2
... ... Office has announced the launch of several innovative new programs to aid ... The first is aimed at entrepreneurs and offers them the opportunity ... 24 months without requiring any equity in their start-up companies. The ...
... , ROCKAWAY, N.J., Dec. 14 The ... , published by the Infusion Nurses Society ... Anthracyclines are a group of chemotherapy medications including daunorubicin, ... treatment of various types of cancer since the early 1970s.(2) ...
... ... Powder Flow Tester – helping Brookfield move from sheet metal to a plastic enclosure ... ... a US-based thermoforming company, has completed the delivery of Brookfield Engineering’s Powder Flow Tester ...
Cached Biology Technology:The Prinz Law Office Launches Innovative New Programs to Aid Cash-Strapped Entrepreneurs and Businesses 2Infusion Nurses Society References Totect(R) (Dexrazoxane For Injection) as the Only Antidote for the Treatment of Anthracycline Extravasation 2Infusion Nurses Society References Totect(R) (Dexrazoxane For Injection) as the Only Antidote for the Treatment of Anthracycline Extravasation 3Infusion Nurses Society References Totect(R) (Dexrazoxane For Injection) as the Only Antidote for the Treatment of Anthracycline Extravasation 4Infusion Nurses Society References Totect(R) (Dexrazoxane For Injection) as the Only Antidote for the Treatment of Anthracycline Extravasation 5Thermofab Helps Brookfield Engineering Move To A Compact Custom Plastic Enclosure 2
...
... Dyad instrument is the most advanced thermal ... world-class thermal performance, the Dyad also boasts ... as point-and-click navigation via touch pad or ... easily and economically expanded to a four-bay ...
...
Kit for testing the ability of a compound to simulate lipolysis in cultured human adipocytes. Kit contains controls, buffers and reagents for detecting glycerol and non-esterified free fatty acids. A...
Biology Products: