CRISPR/Cas9-based disease modelling and functional correction of Interleukin 7 Receptor alpha Severe Combined Immunodeficiency in T-lymphocytes and hematopoietic stem cells
Rajeev Rai, Zohar Steinberg, Marianna Romito, Federica Zinghirino, Yiting Hu, Nathan White, Asma Naseem, Adrian James Thrasher, Giandomenico Turchiano, Alessia Cavazza
The paper develops a CRISPR/Cas9-based gene editing strategy to correct IL7R-SCID in T cells and HSPCs, restoring IL7R expression and T cell development.
Human Gene Therapy · Jan 22, 2024Peer-reviewedMolecular Biology