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Conference Briefings

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

Daniela Cesana, PhD

Clinical Outcomes in Adults With Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data From the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial

Wolfgang A. Miesbach, MD, PhD

Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings

Giuseppe Ronzitti, PhD

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial

Margareth C. Ozelo, MD, PhD

Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond

Alessio Cantore, PhD

Liver Safety Case Report From the Phase 3 HOPE-B Gene Therapy Trial in Adults With Hemophilia B

Steven W. Pipe, MD

Current Update of Clinical trials on Gene Therapy for Hemophilia

Wolfgang Miesbach, MD

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

David Lillicrap, MD

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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