Highlights From the ISTH 2021 Congress
Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model
P. Batty1, B. Yates2, D. Hurlbut3, S. Siso-Llonch2, A. Menard3, H. Akeefe2, T. Christianson2, P. Colosi2, L. Harpell1, S. Liu2, A. Mo1, A. Pender1, G. Veres2, C. Vitelli2, A. Winterborn4, S. Bunting2, S. Fong2, D. Lillicrap1
1Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada
2BioMarin Pharmaceutical, Novato, United States
3Kingston Health Sciences, Kingston, Canada
4Animal Care Services, Queen's University, Kingston, Canada
RELATED CONTENT
Interactive Webinars
Long-term Outcomes: Durability and Safety
Presented by Prof. Margareth C. Ozelo, MD, PhD...
Obstacles and Opportunities
Presented by Frank W.G. Leebeek, MD, PhD...
Patient Support, Patient Counseling, and Monitoring
Presented by Lindsey A. George, MD...
Gene Therapy for FVIII
Presented by K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPC...
Update on Efficacy of Clinical Trials
Presented by Guy Young, MD...
Adeno-Associated Viral (AAV) Vector Gene Therapy: Application to Hemophilia
Presented by Barbara A. Konkle, MD...
Gene Therapy for Treatment of Hemophilia: An Introduction to Adeno-Associated Viral Vector Gene Transfer
Presented by Johnny Mahlangu, BSc, MBBCh, MMed, FCPath...
Gene Therapy for Treatment of Hemophilia: Common Concerns in Gene Therapy
Presented by Thierry VandenDriessche, PhD...
Gene Therapy for Treatment of Hemophilia: Other Strategies and Targets
Presented by Glenn F. Pierce, MD, PhD...
A History of Hemophilia Treatment: Non-replacement Therapy to Gene Therapy
Presented by Steven W. Pipe, MD...
Getting to Know Gene Therapy: Terminology and Concepts
Presented by David Lillicrap, MD...
A History of Hemophilia Treatment: Factor Replacement to Gene Therapy
Presented by Flora Peyvandi, MD, PhD...
Podcasts