ArticleCell2022
Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells.
Article in Cell, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07740499 (Exploring and Exploiting the TOLerogenic Potential of Hematopoietic Stem and Progenitor Cells to Cure Pediatric Inflammatory Bowel Disease), which is not on this map. Cited by 40 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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.
Exploring and Exploiting the TOLerogenic Potential of Hematopoietic Stem and Progenitor Cells to Cure Pediatric Inflammatory Bowel Disease
Who cites it
40 citing papers in PubMed, 54 citations in OpenAlex.
- Tumor-targeted interferon-α gene therapy for glioblastoma: a phase 1 trial.Nature medicine · 2026Trial
- Unlocking the curative potential of gene transfer and editing for hematopoietic disorders.Experimental hematology · 2026Review
- Article
- Article
- Gene editing of hematopoietic stem cells: applications and advances.International journal of hematology · 2026Review
- Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters.Nature biotechnology · 2026Article
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Targeted inhibition ofMolecular therapy. Methods & clinical development · 2025Article
- Expansion characteristics of CD34Human cell · 2025Article
- CRISPR-based therapeutic genome editing for inherited blood disorders.Nature reviews. Drug discovery · 2025Review
- 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
- Mobilization meets antibody conditioning: A path toward safer engraftment.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- GroβT rapidly and reliably mobilizes primitive hematopoietic stem and progenitor cells in nonhuman primates.Molecular therapy. Methods & clinical development · 2025Article
- Article
- A non-genotoxic stem cell therapy boosts lymphopoiesis and averts age-related blood diseases in mice.Nature communications · 2025Article
- Myeloablative Radioligand Therapy Targeting C-X-C Motif Chemokine Receptor 4 in Advanced Multiple Myeloma.Clinical nuclear medicine · 2025Article
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Enhanced engraftment of human haematopoietic stem cells via mechanical remodelling mediated by the corticotropin-releasing hormone.Nature biomedical engineering · 2025Article
- Hematopoietic stem cells: Understanding the mechanisms to unleash the therapeutic potential of hematopoietic stem cell transplantation.Stem cell research & therapy · 2025Review
- Simultaneous Co-transplantation for Highly Efficient Cell Therapy.Current stem cell research & therapy · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo, and infused, after the administration of toxic myeloablative conditioning to deplete the bone marrow (BM) for the modified cells. We show that mobilizers create an opportunity for seamless engraftment of exogenous cells, which effectively outcompete those mobilized, to repopulate the depleted BM. The competitive advantage results from the rescue during ex vivo culture of a detrimental impact of mobilization on HSPCs and can be further enhanced by the transient overexpression of engraftment effectors exploiting optimized mRNA-based delivery. We show the therapeutic efficacy in a mouse model of hyper IgM syndrome and further developed it in human hematochimeric mice, showing its applicability and versatility when coupled with gene transfer and editing strategies. Overall, our findings provide a potentially valuable strategy paving the way to broader and safer use of HSPC-GT.
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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.