Friedreich's Ataxia -- An Effective Gene Therapy In An Animal Model

Friedreich's Ataxia -- An Effective Gene Therapy In An Animal Model. Science 2.0, By News Staff | April 6th 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.

As the central nervous system is also a target of AAV vectors, Hélène 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.