- Review
Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care
- Marian L. Henderson,
- Jacob K. Zieba,
- Xiaopeng Li,
- Daniel B. Campbell,
- Michael R. Williams,
- Daniel L. Vogt,
- Caleb P. Bupp,
- Yvonne M. Edgerly,
- Surender Rajasekaran and
- Jena M. Krueger
- + 2 authors
Gene therapy holds promise as a life-changing option for individuals with genetic variants that give rise to disease. FDA-approved gene therapies for Spinal Muscular Atrophy (SMA), cerebral adrenoleukodystrophy, β-Thalassemia, hemophilia A/B, re...

