Hemophilia gene therapy knowledge and perceptions: Results of an international survey.

Flora Peyvandi David LillicrapJohnny MahlanguClaire McLintockK John PasiSteven W PipeWendy ScalesAlok SrivastavaThierry VandenDriessche

Published in: Research and practice in thrombosis and haemostasis (2020)

This survey highlighted knowledge gaps and educational needs related to gene therapy for hemophilia and, along with other inputs, has informed the development of "Gene Therapy in Hemophilia: An ISTH Education Initiative."

Keyphrases

gene therapy
healthcare
quality improvement
cross sectional
genome wide
copy number
transcription factor
drug induced