Advanced genetic engineering of hematopoiesis

Pubblicazioni Scientifiche

• CELL

  Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells

• CELL STEM CELL

  Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells

• CELL STEM CELL

  In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease

• EMBO MOLECULAR MEDICINE

  Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy

• EMBO MOLECULAR MEDICINE

  Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives

• NATURE BIOTECHNOLOGY

  Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

• NATURE PROTOCOLS

  BAR-Seq clonal tracking of gene-edited cells

• SCIENCE TRANSLATIONAL MEDICINE

  Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency