Free to Access: New Special Report on Gene Therapy

 

Innovations In: Gene therapy

Gene therapy

Gene therapy has come a long way since its first proof-of-concept experiments in the 1990s. The approach - which involves fixing or replacing a disease-causing gene or changing its activity - has recorded some remarkable successes and some devastating missteps. In the past decade those extreme ups and downs have leveled off and now gene therapy, in a variety of forms, has begun advancing at rapid pace. This special Innovations In explores: How the field has moved beyond its early failures and grown to encompass an ever expanding vision of what genomic medicine is and what it can accomplish; How researchers are finding ways to minimize the risk of serious side effects that plagued the discipline’s early years; Four success stories that demonstrate how gene therapy is beginning to fulfil its potential and that offer a glimpse of what is to come; Why researchers, practitioners and patients must have open conversations to balance gene therapy’s promise with its reality.

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