
We Need to Ground Truth Assumptions about Gene Therapy
Researchers, practitioners and patients must balance the discipline’s promise with its reality
After facing early challenges, the field is moving toward the medical mainstream
This special report is editorially independent and was produced with financial support from Pfizer.

Luisa Jung

We Need to Ground Truth Assumptions about Gene Therapy
Researchers, practitioners and patients must balance the discipline’s promise with its reality

Four Success Stories in Gene Therapy
The field is beginning to fulfill its potential. These therapies offer a glimpse of what’s to come

From Discovery Through Delivery: Patient-Focused Development of Gene Therapies For Rare Diseases

Gene Therapy Is Coming of Age
Various approaches are approved for treating blood cancers and a few rare disorders—they may soon become standard care

The Definition of Gene Therapy Has Changed
Over the past few years, the discipline has evolved in significant ways

The Quest to Overcome Gene Therapy’s Failures
Tragic side effects plagued the field’s early years, but researchers are finding ways to minimize the risks