Mobilization-based engraftment of haematopoietic stem cells: a new perspective for chemotherapy-free gene therapy and transplantation

Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

(2023) Samuele Ferrari et al.   Cell Stem Cell

Chemically defined cytokine-free expansion of human haematopoietic stem cells

(2023) Masatoshi Sakurai et al.   NATURE

Defibrotide plus best standard of care compared with best standard of care alone for the prevention of sinusoidal obstruction syndrome (HARMONY): a randomised, multicentre, phase 3 trial

(2023) Stephan A Grupp et al.   Lancet Haematology

Basic characteristics and safety of donation in related and unrelated haematopoietic progenitor cell donors – first 10 years of prospective donor follow-up of Swiss donors

(2022) M. Rüesch et al.   BONE MARROW TRANSPLANTATION

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

(2022) Attya Omer-Javed et al.   CELL

Worked to the bone: antibody-based conditioning as the future of transplant biology

(2022) James M. Griffin et al.   Journal of Hematology & Oncology

A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

(2022) Francesca Tucci et al.   Nature Communications

The cellular composition and function of the bone marrow niche after allogeneic hematopoietic cell transplantation

(2022) Flavia Peci et al.   BONE MARROW TRANSPLANTATION

Targeted inducible delivery of immunoactivating cytokines reprograms glioblastoma microenvironment and inhibits growth in mouse models

(2022) Filippo Birocchi et al.   Science Translational Medicine

In vivo HSC transduction in rhesus macaques with an HDAd5/3+ vector targeting desmoglein 2 and transiently overexpressing cxcr4

(2022) Hongjie Wang et al.   Blood Advances

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

(2021) Valentina Vavassori et al.   EMBO Molecular Medicine

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

(2021) Chang Li et al.   Blood Advances

Niches that regulate stem cells and hematopoiesis in adult bone marrow

(2021) Stefano Comazzetto et al.   DEVELOPMENTAL CELL

Myeloid cell‐based delivery of IFN‐γ reprograms the leukemia microenvironment and induces anti‐tumoral immune responses

(2021) Adele Mucci et al.   EMBO Molecular Medicine

Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series

(2021) Daniele Canarutto et al.   Molecular Therapy-Methods & Clinical Development

Mobilization‐based transplantation of young‐donor hematopoietic stem cells extends lifespan in mice

(2020) Michael J. Guderyon et al.   AGING CELL

Characteristics of cells with engraftment capacity within CD34+ cell population upon G-CSF and Plerixafor mobilization

(2020) Margaux Mombled et al.   LEUKEMIA

History of hematopoietic cell transplantation: challenges and progress

(2020) Noa Granot et al.   HAEMATOLOGICA

Gene therapy using haematopoietic stem and progenitor cells

(2020) Giuliana Ferrari et al.   NATURE REVIEWS GENETICS

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice

(2019) Hye-Sook Kwon et al.   BLOOD

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

(2019) Luigi Naldini   EMBO Molecular Medicine

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

(2019) Agnieszka Czechowicz et al.   Nature Communications

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

(2019) Giulia Schiroli et al.   Cell Stem Cell

Long-term ex vivo haematopoietic-stem-cell expansion allows nonconditioned transplantation

(2019) Adam C. Wilkinson et al.   NATURE

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

(2019) Paula Río et al.   NATURE MEDICINE

Non-toxic HSC Transplantation-Based Macrophage/Microglia-Mediated GDNF Delivery for Parkinson’s Disease

(2019) Cang Chen et al.   Molecular Therapy-Methods & Clinical Development

Differences in Cellular Composition of Peripheral Blood Stem Cell Grafts from Healthy Stem Cell Donors Mobilized with Either Granulocyte Colony-Stimulating Factor (G-CSF) Alone or G-CSF and Plerixafor

(2018) Raphael Teipel et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

(2018) Hongjie Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

Late effects of blood and marrow transplantation

(2017) Yoshihiro Inamoto et al.   HAEMATOLOGICA

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

(2017) Giulia Schiroli et al.   Science Translational Medicine

Late effects of blood and marrow transplantation

(2017) Yoshihiro Inamoto et al.   HAEMATOLOGICA

In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors

(2016) M. Richter et al.   BLOOD

Phase I/II Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft

(2016) John E. Wagner et al.   Cell Stem Cell

Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to plerixafor alone

(2016) Maria Rosa Lidonnici et al.   HAEMATOLOGICA

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin

(2016) Rahul Palchaudhuri et al.   NATURE BIOTECHNOLOGY

Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to plerixafor alone

(2016) Maria Rosa Lidonnici et al.   HAEMATOLOGICA

Leukemia cell mobilization with G-CSF plus plerixafor during busulfan–fludarabine conditioning for allogeneic stem cell transplantation

(2015) M Konopleva et al.   BONE MARROW TRANSPLANTATION

Cytoreductive conditioning for severe combined immunodeficiency – help or hindrance?

(2015) Alexandra Laberko et al.   Expert Review of Clinical Immunology

Conditioning regimens for hematopoietic cell transplantation: one size does not fit all

(2014) B. Gyurkocza et al.   BLOOD

Kit Regulates HSC Engraftment across the Human-Mouse Species Barrier

(2014) Kadriye Nehir Cosgun et al.   Cell Stem Cell

Mobilization of healthy donors with plerixafor affects the cellular composition of T-cell receptor (TCR)-αβ/CD19-depleted haploidentical stem cell grafts

(2014) Sergio Rutella et al.   Journal of Translational Medicine

A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency

(2014) Christopher C. Dvorak et al.   PEDIATRIC TRANSPLANTATION

Differences in the Phenotype, Cytokine Gene Expression Profiles, and In Vivo Alloreactivity of T Cells Mobilized with Plerixafor Compared with G-CSF

(2013) A. Lundqvist et al.   JOURNAL OF IMMUNOLOGY

Hematopoietic stem cell mobilization: updated conceptual renditions

(2012) H Bonig et al.   LEUKEMIA

Mobilization of hematopoietic stem and progenitor cells using inhibitors of CXCR4 and VLA-4

(2011) M P Rettig et al.   LEUKEMIA

BIO5192, a small molecule inhibitor of VLA-4, mobilizes hematopoietic stem and progenitor cells

(2009) P. Ramirez et al.   BLOOD

Hematopoietic stem cell transplantation without irradiation

(2009) Claudia Waskow et al.   NATURE METHODS

Rapid mobilization of functional donor hematopoietic cells without G-CSF using AMD3100, an antagonist of the CXCR4/SDF-1 interaction

(2008) S. M. Devine et al.   BLOOD