ArticleBlood2020
The BET inhibitor CPI203 promotes ex vivo expansion of cord blood long-term repopulating HSCs and megakaryocytes.
Article in Blood, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 27 citations in OpenAlex.
- Current understanding and new promises of umbilical cord blood in emerging clinical applications.Science China. Life sciences · 2026Review
- Ex vivo expansion of hematopoietic stem and progenitor cells from human mobilized peripheral blood for gene therapy applications.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Transient mechanical activation of the Piezo1 channel facilitates ex vivo expansion of hematopoietic stem cells.Cell research · 2026Article
- Hematopoietic Stem Cell Aging: Mechanisms, Microenvironment Influences, and Rejuvenation Strategies.Bioengineering (Basel, Switzerland) · 2025Review
- Deciphering the Complexities of Adult Human Steady State and Stress-Induced Hematopoiesis: Progress and Challenges.International journal of molecular sciences · 2025Review
- Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy.Blood advances · 2024Article
- Ex vivo expansion of human hematopoietic stem cells and clinical applications.Cancer science · 2024Review
- Advances in ex vivo expansion of hematopoietic stem and progenitor cells for clinical applications.Frontiers in bioengineering and biotechnology · 2024Review
- Expansion of human hematopoietic stem cells by inhibiting translation.bioRxiv : the preprint server for biology · 2023Article
- Engineered hematopoietic and immune cells derived from human pluripotent stem cells.Experimental hematology · 2023Review
- Review
- The quest for the holy grail: overcoming challenges in expanding human hematopoietic stem cells for clinical use.Stem cell investigation · 2023Review
- Ex Vivo Expansion and Homing of Human Cord Blood Hematopoietic Stem Cells.Advances in experimental medicine and biology · 2023Article
- Clinical Progress and Preclinical Insights Into Umbilical Cord Blood Transplantation Improvement.Stem cells translational medicine · 2022Review
- Phase Ib Study of the BET Inhibitor GS-5829 as Monotherapy and Combined with Enzalutamide in Patients with Metastatic Castration-Resistant Prostate Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2022Article
- Development and clinical advancement of small molecules forActa pharmaceutica Sinica. B · 2022Review
- Increasing Complexity of Molecular Landscapes in Human Hematopoietic Stem and Progenitor Cells during Development and Aging.International journal of molecular sciences · 2022Review
- Development of LT-HSC-Reconstituted Non-Irradiated NBSGW Mice for the Study of Human HematopoiesisFrontiers in immunology · 2021Article
- Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?Frontiers in genome editing · 2020Review
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Authors and funding
15 authors at 3 institutions in 3 countries.
Funding
Abstract
Although cytokine-mediated expansion of human hematopoietic stem cells (HSCs) can result in high yields of hematopoietic progenitor cells, this generally occurs at the expense of reduced bone marrow HSC repopulating ability, thereby limiting potential therapeutic applications. Because bromodomain-containing proteins (BCPs) have been demonstrated to regulate mouse HSC self-renewal and stemness, we screened small molecules targeting various BCPs as potential agents for ex vivo expansion of human HSCs. Of 10 compounds tested, only the bromodomain and extra-terminal motif inhibitor CPI203 enhanced the expansion of human cord blood HSCs without losing cell viability in vitro. The expanded cells also demonstrated improved engraftment and repopulation in serial transplantation assays. Transcriptomic and functional studies showed that the expansion of long-term repopulating HSCs was accompanied by synchronized expansion and maturation of megakaryocytes consistent with CPI203-mediated reprogramming of cord blood hematopoietic stem and progenitor cells. This approach may therefore prove beneficial for ex vivo gene editing, for enhanced platelet production, and for the improved usage of cord blood for transplantation research and therapy.
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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.