Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII
Summary
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. Copyright © 2022 Ramamurthy, Rodriguez, Ainsworth, Shields, Meares, Bishop, Farland, Langefeld, Atala, Doering, Spencer, Porada and Almeida-Porada.
| Authors | Ramamurthy RM, Rodriguez M, Ainsworth HC, Shields J, Meares D, Bishop C, Farland A, Langefeld CD, Atala A, Doering CB, Spencer HT, Porada CD, Almeida-Porada G |
|---|---|
| Journal | Frontiers in immunology |
| Publication Date | 2022;13:954984 |
| PubMed | 36591257 |
| PubMed Central | PMC9800010 |
| DOI | 10.3389/fimmu.2022.954984 |