Advanced genetic engineering of hematopoiesis

Pubblicazioni Scientifiche

• 2022-06-23 CELL

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

• 2018-12-06 CELL STEM CELL

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

• 2019-11-07 CELL STEM CELL

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

• 2017-11-01 EMBO MOLECULAR MEDICINE

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

• 2019-03-01 EMBO MOLECULAR MEDICINE

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

• 2020-11-01 NATURE BIOTECHNOLOGY

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

• 2021-06-01 NATURE PROTOCOLS

  BAR-Seq clonal tracking of gene-edited cells

• 2024-02-07 SCIENCE TRANSLATIONAL MEDICINE

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