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

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

IntroductionPlacenta-derived mesenchymal cells (PLCs) endogenously produce FVIII, which makes them ideally suited for cell-based fVIII gene delivery. We have previously reported that human PLCs can be efficiently modified with a lentiviral vector encoding a bioengineered, expression/secretion-optimi...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-12-01
Series:Frontiers in Immunology
Subjects:
CRISPR/Cas
lentiviral (LV) vector
cell therapy
gene therapy
FVIII
hemophilia A
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2022.954984/full

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https://www.frontiersin.org/articles/10.3389/fimmu.2022.954984/full

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