Gene correction of CD40LG gene in T cells and HSPC for the treatment of X-linked hyper-IgM immunodeficiency

Pubblicazioni Scientifiche

• EMBO MOLECULAR MEDICINE

  Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome

• FRONTIERS IN GENOME EDITING

  Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation

• NATURE PROTOCOLS

  BAR-Seq clonal tracking of gene-edited cells

• SCIENCE TRANSLATIONAL MEDICINE

  Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1