The Gene Fix

THREE DECADES AFTER ITS FIRST, faltering steps in humans, gene therapy is emerging as a treatment option for a small but growing number of diseases. Although the concept faced scientific and ethical uncertainty when it was floated in the 1970s, the foundation of the ap-proach-replacing or fixing a single, disease-causing gene-has proved solid. Researchers have developed different ways to correct or influence the way someone's genes function and used those techniques to create therapies for several blood disorders, as well as degenerative eye and muscle diseases. More than half a dozen such treatments have gained approval in the U.S. in the past five years, and numerous others, aimed at a variety of conditions, are progressing toward clinical trials.

Automated summary

Gene therapy has emerged as a treatment option for various diseases after three decades of development. Replacing or fixing disease-causing genes has been proven effective, with treatments available for blood disorders, eye diseases, and muscle diseases. Over half a dozen gene therapy treatments have gained approval in the U.S. in the past five years, with more progressing towards clinical trials for a range of conditions.