ArticleBlood2019
Anti-CD117 antibody depletes normal and myelodysplastic syndrome human hematopoietic stem cells in xenografted mice.
Article in Blood, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers.
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Who cites it
50 citing papers in PubMed, 71 citations in OpenAlex.
- Antimetabolites synergize with non-genotoxic antibody drug conjugate conditioning in hematopoietic stem cell lentiviral gene therapy.Molecular therapy. Advances · 2026Article
- Immune reset: what do we learn from bone marrow transplantation.Medical review (2021) · 2026Review
- CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model.bioRxiv : the preprint server for biology · 2026Article
- Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- CAR T-cells targeting CD117 effectively eliminate mast cells in preclinical models of advanced systemic mastocytosis.Leukemia · 2026Article
- Preventing graft re-JAK-tion: safer transplant conditioning enables murine islet allograft tolerance and diabetes reversal.The Journal of clinical investigation · 2026Article
- Gene therapy for inborn errors of immunity: past progress, current status and future directions.Rare disease and orphan drugs journal · 2025Article
- To c or not to c-KIT?: High toxicity and modest efficacy in CD117-directed CAR T cell therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- HSC engraftment is enhanced by combining mobilization with anti-C-Kit and Anti-CD47-based conditioning in hematopoietic transplant.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial.Nature medicine · 2025Article
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- Development of multivalent CAR T cells as dual immunotherapy and conditioning agents.Molecular therapy. Oncology · 2025Article
- Next generation targeted non-genotoxic conditioning for hematopoietic stem cell and hematopoietic stem cell-based gene therapy.Frontiers in immunology · 2025Review
- Efficient combinatorial adaptor-mediated targeting of acute myeloid leukemia with CAR T-cells.Leukemia · 2024Article
- Article
- Targeted hematopoietic stem cell depletion through SCF-blockade.Stem cell research & therapy · 2024Article
- Targeting CD45 by gene-edited CAR T cells for leukemia eradication and hematopoietic stem cell transplantation preconditioning.Molecular therapy. Oncology · 2024Article
- Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Review
Corrections and comments
- Commented on bySelective CD1172019
Authors and funding
8 authors at 6 institutions in 2 countries.
Funding
Abstract
The myelodysplastic syndromes (MDS) represent a group of clonal disorders that result in ineffective hematopoiesis and are associated with an increased risk of transformation into acute leukemia. MDS arises from hematopoietic stem cells (HSCs); therefore, successful elimination of MDS HSCs is an important part of any curative therapy. However, current treatment options, including allogeneic hematopoietic cell transplantation (HCT), often fail to ablate disease-initiating MDS HSCs, and thus have low curative potential and high relapse rates. Here, we demonstrate that human HSCs can be targeted and eliminated by monoclonal antibodies (mAbs) that bind cell-surface CD117 (c-Kit). We show that an anti-human CD117 mAb, SR-1, inhibits normal cord blood and bone marrow HSCs in vitro. Furthermore, SR-1 and clinical-grade humanized anti-human CD117 mAb, AMG 191, deplete normal and MDS HSCs in vivo in xenograft mouse models. Anti-CD117 mAbs also facilitate the engraftment of normal donor human HSCs in MDS xenograft mouse models, restoring normal human hematopoiesis and eradicating aggressive pathologic MDS cells. This study is the first to demonstrate that anti-human CD117 mAbs have potential as novel therapeutics to eradicate MDS HSCs and augment the curative effect of allogeneic HCT for this disease. Moreover, we establish the foundation for use of these antibody agents not only in the treatment of MDS but also for the multitude of other HSC-driven blood and immune disorders for which transplant can be disease-altering.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.