Lentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial

Abstract

	•This was the first gene therapy clinical trial to be completed for Fabry disease.
	•There were no adverse events of any grade attributable to the cellular gene therapy intervention or host conditioning throughout the follow-up interval of 5 years.
	•After reduced-intensity melphalan treatment, all patients engrafted their autologous modified α-gal A expressing cells.
	•All patients synthesized and secreted α-gal A throughout the course of the study.
	•Expression of α-gal A resulted in a decrease in plasma lyso-Gb3 in four of five patients and stabilization of kidney symptoms in all patients.

Keywords

clinical trial results / haematopoietic stem cells / lysosomal storage disorders / melphalan conditioning / progenitor cells

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