Frataxin Restoration in the Nervous System: Possibilities for Gene Therapy

David R. Lynch, Elizabeth Kichula, and Hong Lin. Molecular Therapy, Available online 30 June 2018, ISSN 1525-0016, doi:10.1016/j.ymthe.2018.06.006

Many obstacles beyond those identified in the present study will appear in the translation of gene therapy from animal concept to human therapy. Still, the present work provides proof that gene therapy may be useful long term in FRDA, and hope for all those with this disorder.

 Frataxin Restoration in the Nervous System: Possibilities for Gene Therapy