Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review.

Cihan AyLaurent FrenzelKaren PinachyanSandra Le Quellec

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2023)

This illustrated review educates healthcare professionals on AAV-based gene therapy for haemophilia A and B enabling them to further educate their peers and their patients.

Keyphrases

gene therapy
end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
prognostic factors
copy number
gene expression
patient reported
dna methylation
transcription factor
genome wide identification