From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World

From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World . John J.P. Kastelein, Colin J.D. Ross, and Michael R. Hayden. Human Gene Therapy. May 2013, 24(5): 472-478. doi:10.1089/hum.2013.063.

We hope soon have one similar paper for Friedreich's ataxia ....