ISTH 2023 | The future generations of gene therapy for hemophilia
Radek Kaczmarek, PhD, Indiana University School of Medicine, Indianapolis, IN, discusses the current state of gene therapies for hemophilia, as well as the improvements which are being made in pre-clinical studies. Adeno-associated virus (AAV)-based gene therapies represent progress in the treatment of hemophilia, but are not without imperfections, including gene dilution and variability in efficacy. Dr Kaczmarek discusses novel approaches being investigated which can overcome these issues in the future. This interview took place at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), held in Montréal, Canada.
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