Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII.

Ritu M Ramamurthy,Martin Rodriguez,Hannah C Ainsworth,Jordan Shields,Diane Meares,Colin Bishop,Andrew Farland,Carl D Langefeld, Anthony Atala,Christopher B Doering,H Trent Spencer,Christopher D Porada,Graça Almeida-Porada

Frontiers in immunology（2022）

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Abstract

Although we did not achieve our primary objective, our results validate the utility of both PLCs and hLSECs as cell-based delivery vehicles for a fVIII transgene, and they highlight the hurdles that remain to be overcome before primary human cells can be gene-edited with sufficient efficiency for use in cell-based gene therapy to treat HA.

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Key words

CRISPR/Cas,FVIII,cell therapy,gene therapy,hemophilia A,lentiviral (LV) vector,placental-derived mesenchymal stromal cells

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