Gene correction of CD40LG gene in T cells and HSPC for the treatment of X-linked hyper-IgM immunodeficiency
- 5 Anni 2016/2021
- 491.603€ Totale Fondi
Descrizione non disponibile
Pubblicazioni Scientifiche
- 2021-03-05 EMBO MOLECULAR MEDICINE
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome
- 2021-03-31 FRONTIERS IN GENOME EDITING
Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation
- 2021-06-01 NATURE PROTOCOLS
BAR-Seq clonal tracking of gene-edited cells
- 2017-10-11 SCIENCE TRANSLATIONAL MEDICINE
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
