The Forever Fix: Gene Therapy and the Boy Who Saved It

In this impressive, meticulously researched study of the exciting new developments in gene therapy, geneticist and journalist Lewis (Human Genetics) looks closely at the history of setbacks plaguing the treatment of rare genetic diseases as well as recent breakthroughs. The success in reversing eight-year-old Corey Haas’s blindness from the rare inherited disorder Leber congenital amaurosis by gene therapy in 2008 has proved the “happy story” that the field needed after the well-publicized death in 1999 of Jesse Gelsinger after gene therapy to treat his inherited metabolic deficiency. Case by case Lewis chronicles the small steps in advancing treatment of rare disorders of the very young, such as severe combined immune deficiency, adrenoleukodystrophy, giant axonal neuropathy, and Canavan and Tay-Sachs diseases afflicting the Ashkenazi Jewish community—all of which have undergone years of trial and error and benefited from the advocacy of parents. Lewis traces the evolution of the idea of gene therapy by William French Anderson from the 1950s, leading to approaches in recombinant DNA technology in the 1970s with animal testing, and culminating in the first sequencing of the human genome in 2000. Issues of patent-control of certain genes inhibit testing while large pharmaceutical companies continue to drag their heels in funding research. Yet with each success, as Lewis recounts in this rigorous, energetic work, possibilities in treating HIV infection and dozens of other diseases might be around the next corner. Agent: Ellen Geiger. (Feb.)