ArticleBlood2016
Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates.
Article in Blood, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
48 citing papers in PubMed.
- Cancer neuroscience: signaling pathways and new therapeutic strategies for cancer.Signal transduction and targeted therapy · 2026Review
- Engraftment and persistence ofScience translational medicine · 2025Article
- Protection of CD33-modified hematopoietic stem cell progeny from CD33-directed CAR T cells in rhesus macaques.Blood advances · 2025Article
- In pursuit of an HIV cure: from stem cell transplants to gene therapies.Frontiers in genome editing · 2025Review
- Restoring T and B cell generation in X-linked severe combined immunodeficiency mice through hematopoietic stem cells adenine base editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Efficient manufacturing and engraftment ofMolecular therapy. Methods & clinical development · 2023Article
- mRNA therapeutics: New vaccination and beyond.Fundamental research · 2023Review
- Identification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Review
- mRNA-based therapeutics: powerful and versatile tools to combat diseases.Signal transduction and targeted therapy · 2022Review
- Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells.Molecular therapy. Methods & clinical development · 2022Article
- Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges.Science translational medicine · 2022Review
- Nonhuman Primates in Translational Research.Annual review of animal biosciences · 2022Review
- Identification of the minimum requirements for successful haematopoietic stem cell transplantation.British journal of haematology · 2022Article
- Promising Stem Cell therapy in the Management of HIV and AIDS: A Narrative Review.Biologics : targets & therapy · 2022Review
- Gene Editing for Inherited Red Blood Cell Diseases.Frontiers in physiology · 2022Review
- Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Genome editing in large animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- The Strategies and Challenges of CCR5 Gene Editing in Hematopoietic Stem and Progenitor Cells for the Treatment of HIV.Stem cell reviews and reports · 2021Review
- Broadly active zinc finger protein-guided transcriptional activation of HIV-1.Molecular therapy. Methods & clinical development · 2021Article
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.
Funding
Abstract
Genome editing in hematopoietic stem and progenitor cells (HSPCs) is a promising novel technology for the treatment of many human diseases. Here, we evaluated whether the disruption of the C-C chemokine receptor 5 (CCR5) locus in pigtailed macaque HSPCs by zinc finger nucleases (ZFNs) was feasible. We show that macaque-specific CCR5 ZFNs efficiently induce CCR5 disruption at levels of up to 64% ex vivo, 40% in vivo early posttransplant, and 3% to 5% in long-term repopulating cells over 6 months following HSPC transplant. These genome-edited HSPCs support multilineage engraftment and generate progeny capable of trafficking to secondary tissues including the gut. Using deep sequencing technology, we show that these ZFNs are highly specific for the CCR5 locus in primary cells. Further, we have adapted our clonal tracking methodology to follow individual CCR5 mutant cells over time in vivo, reinforcing that CCR5 gene-edited HSPCs are capable of long-term engraftment. Together, these data demonstrate that genome-edited HSPCs engraft, and contribute to multilineage repopulation after autologous transplantation in a clinically relevant large animal model, an important step toward the development of stem cell-based genome-editing therapies for HIV and potentially other diseases as well.
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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.