In mice, 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. These findings are published in iNature Medicine/i 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 ...Monday 7 April 2014
Friedreich's Ataxia - an Effective Gene Therapy in Mice Model
In mice, 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. These findings are published in iNature Medicine/i 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 ...
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