ISTH 2023 | Current hurdles in AAV-based gene therapy for hemophilia
Radek Kaczmarek, PhD, Indiana University School of Medicine, Indianapolis, IN, discusses the challenges associated with adeno-associated virus (AAV)-based gene therapy for patients with hemophilia. AAV gene therapies are currently the only treatment available with the potential to allow patients with hemophilia to become treatment-independent. Dr Kaczmarek shares the current uncertainties surrounding the efficacy and safety of these treatments, highlighting the need to conduct more basic research to better understand these therapies. 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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