ArticleThe Journal of experimental medicine2023
Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy.
Article in The Journal of experimental medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
25 citing papers in PubMed, 34 citations in OpenAlex.
- Editing around the target: epitope engineering to protect stem cell grafts.Blood advances · 2026Review
- CRISPR application in hematological disorders: from bench to bedside.Blood advances · 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
- Emerging trends in gene and cell therapy: CRISPR in DNA editing and beyond.Cell reports. Medicine · 2026Review
- Next-generation CAR-T therapy for acute myeloid leukemia: bridging innovation with clinical translation.Annals of hematology · 2026Review
- Antigen-directed p53 restoration inFrontiers in oncology · 2026Article
- SEED-Selection enables high-efficiency enrichment of primary T cells edited at multiple loci.Nature biotechnology · 2025Article
- Targeting the roots of myeloid malignancies with T cell receptors.Nature reviews. Cancer · 2025Review
- Strategic innovations: Tackling challenges of immunotherapy in acute myeloid leukemia.Chinese journal of cancer research = Chung-kuo yen cheng yen chiu · 2025Article
- Engraftment and persistence ofScience translational medicine · 2025Article
- T cells in cancer: mechanistic insights and therapeutic advances.Biomarker research · 2025Review
- Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy.Nature communications · 2025Article
- Precision epitope editing: A path to advanced immunotherapies.Cell insight · 2025Review
- Improving efficacy of in vivo therapy of sickle cell disease by hijacking natural biology of hematopoietic stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Article
- Auto-expansion ofMolecular therapy. Methods & clinical development · 2024Article
- T cell-redirecting therapies in hematological malignancies: Current developments and novel strategies for improved targeting.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Review
- Enhancing cellular immunotherapies in cancer by engineering selective therapeutic resistance.Nature reviews. Cancer · 2024Review
- Review
- Biomimetic Scaffolds-A Novel Approach to Three Dimensional Cell Culture Techniques for Potential Implementation in Tissue Engineering.Nanomaterials (Basel, Switzerland) · 2024Review
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Authors and funding
34 authors at 7 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Targeted eradication of transformed or otherwise dysregulated cells using monoclonal antibodies (mAb), antibody-drug conjugates (ADC), T cell engagers (TCE), or chimeric antigen receptor (CAR) cells is very effective for hematologic diseases. Unlike the breakthrough progress achieved for B cell malignancies, there is a pressing need to find suitable antigens for myeloid malignancies. CD123, the interleukin-3 (IL-3) receptor alpha-chain, is highly expressed in various hematological malignancies, including acute myeloid leukemia (AML). However, shared CD123 expression on healthy hematopoietic stem and progenitor cells (HSPCs) bears the risk for myelotoxicity. We demonstrate that epitope-engineered HSPCs were shielded from CD123-targeted immunotherapy but remained functional, while CD123-deficient HSPCs displayed a competitive disadvantage. Transplantation of genome-edited HSPCs could enable tumor-selective targeted immunotherapy while rebuilding a fully functional hematopoietic system. We envision that this approach is broadly applicable to other targets and cells, could render hitherto undruggable targets accessible to immunotherapy, and will allow continued posttransplant therapy, for instance, to treat minimal residual disease (MRD).
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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.