Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease.

Ju Huang, Aneal Khan, Bryan C Au, Dwayne L Barber, Lucía López-Vásquez, Nicole L Prokopishyn, Michel Boutin, Michael Rothe, Jack W Rip, Mona Abaoui, Murtaza S Nagree, Shaalee Dworski, Axel Schambach, Armand Keating, Michael L West, John Klassen, Patricia V Turner, Sandra Sirrs, C Anthony Rupar, Christiane Auray-Blais, Ronan Foley, Jeffrey A Medin
Mol Ther Methods Clin Dev 2017 Jun 12;5:241-258. Epub 2017 May 12.

Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34+ hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. ….

–Source: https://www.ncbi.nlm.nih.gov/pubmed/28603745