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

ravenous injection of AAVrh10 expressing frataxin is not only capable of preventing the development of heart disease in animals before the appearance of symptoms, but also, more impressively, of fully and rapidly curing the hearts of animals at an advanced stage of heart disease. After three weeks of treatment, the heart become fully functional again; mitochondrial function and the appearance of heart tissue being very similar to those of healthy mice. "This is the first time that gene therapy has prompted full, lasting remission of heart disease so quickly in an animal model." explains Hlne Puccio.

As the central nervous system is also a target of AAV vectors, Hlne Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

Based on these promising results, work has begun on the necessary developments to propose to patients suffering from Friedreich's ataxia and presenting a progressive cardiomyopathy a treatment by gene therapy. To this end, three of the paper's authors have set up AAVLife, a French company specialising in gene therapy for rare diseases, to translate to clinic these important laboratory findings. A patent application has been submitted by Inserm Transfert for this gene therapy approach.


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

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Friedreich's ataxia -- an effective gene therapy in an animal model
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