Towards a one-time cure for Fabry disease: Lentivirus-mediated haematopoietic stem and progenitor cell gene therapy

Rina Kansal

doi:10.1002/ctd2.70042

Clinical and Translational Discovery ›› 2025, Vol. 5 ›› Issue (2) : e70042 DOI: 10.1002/ctd2.70042

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Towards a one-time cure for Fabry disease: Lentivirus-mediated haematopoietic stem and progenitor cell gene therapy

Rina Kansal ¹^,²^,^†

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Keywords

α-galactosidase A deficiency disease / clinical trial / Fabry–Anderson disease / gene therapy / genetic disease / haematopoietic cells / inborn error / lysosome storage disorders / metabolism

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Rina Kansal. Towards a one-time cure for Fabry disease: Lentivirus-mediated haematopoietic stem and progenitor cell gene therapy. Clinical and Translational Discovery, 2025, 5(2): e70042 DOI:10.1002/ctd2.70042

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References

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[6]	MedinJA, TudorM, SimovitchR, et al. Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector. Proc Natl Acad Sci U S A. 1996;93(15):7917-7922.

[7]	HuangJ, KhanA, AuBC, et al. Lentivector iterations and pre-clinical scale-up/toxicity testing: targeting mobilized CD34+ cells for correction of Fabry disease. Mol Ther Methods Clin Dev. 2017;5:241-258.

[8]	KhanA, BarberDL, McKillopWM, et al. Lentivirus-mediated gene therapy for Fabry disease:5-year end-of-study results from the Canadian FACTs trial. Clin Transl Med. 2025;15(1):e70073.

[9]	KhanA, BarberDL, HuangJ, et al. Lentivirus-mediated gene therapy for Fabry disease. Nat Commun. 2021;12(1):1178.

[10]	SalehAH, RotheM, BarberDL, et al. Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease. Mol Ther Methods Clin Dev. 2023;28:262-271.

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2025 The Author(s). Clinical and Translational Discovery published by John Wiley & Sons Australia, Ltd on behalf of Shanghai Institute of Clinical Bioinformatics.