Additional Resources
To learn more about gene therapy and rare disease, visit these resources:
- The American Society of Gene & Cell Therapy
- Gene Therapy 101 from ASGCT and NORD (1-hour video)
- Viral Vector Overview (from ASGCT) (5-min video)
- MedlinePlus – Genes and Gene Therapy
- National Center for Advancing Translational Studies (NCATS) - Genetic and Rare Diseases (GARD) Information Center
- National Human Genome Research Institute
- National Organization for Rare Disorders (NORD)
- Orphan Disease Center - Resources
- U.S. Food and Drug Administration (FDA) – Gene Therapy Inside Out (1.5-minute video)