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Gene Therapy Approach to Friedreich’s Ataxia Improve Cardiac Function in Mouse Study
A gene therapy approach to increase the amount of the critical frataxin protein shows that correcting only half of the heart muscle cells, or cardiomyocytes, is enough to fully restore the cardiac function in a mouse model of Friedreich’s Ataxia (FA).
The study, “Correction of half the cardiomyocytes fully rescue Friedreich Ataxia mitochondrial cardiomyopathy through cell-autonomous mechanisms,” was published in the journal Human Molecular Genetics. Click here to read more about the study and its findings.
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