ArticleMolecular therapy. Nucleic acids2018
Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting.
Article in Molecular therapy. Nucleic acids, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 69 papers, 1 of them a synthesis that pooled it.
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.
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Who cites it
69 citing papers in PubMed, 1 synthesis or guideline pooled it, 112 citations in OpenAlex.
- Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta-analysis of preclinical studies.Stem cells translational medicine · 2021Pooled it
- High efficiency CRISPR knock-in demonstrates that TCF1 is insufficient to reverse T cell exhaustion.Nature communications · 2026Article
- Novel humanized loss-of-function NF1 mouse model of juvenile myelomonocytic leukemia.Blood advances · 2026Article
- Harnessing DNA barcoding to enhance and sustain polyclonality in gene-edited hematopoietic stem cells.Molecular therapy. Methods & clinical development · 2025Article
- Distinct and convergent effects ofProceedings of the National Academy of Sciences of the United States of America · 2025Article
- Removal of TREX1 activity enhances CRISPR-Cas9-mediated homologous recombination.Nature biotechnology · 2025Article
- Senescence and inflammation are unintended adverse consequences of CRISPR-Cas9/AAV6-mediated gene editing in hematopoietic stem cells.Cell reports. Medicine · 2025Article
- Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival.Molecular therapy. Nucleic acids · 2025Article
- Generating allogeneic CAR-NKT cells for off-the-shelf cancer immunotherapy with genetically engineered HSP cells and feeder-free differentiation culture.Nature protocols · 2025Review
- Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors.Nature biotechnology · 2025Article
- Allogeneic CD33-directed CAR-NKT cells for the treatment of bone marrow-resident myeloid malignancies.Nature communications · 2025Article
- Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis.Nature communications · 2025Article
- Therapeutic gene correction of HBB frameshift CD41-42 (-TCTT) deletion in human hematopoietic stem cells.Advanced biotechnology · 2025Article
- Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells.Nature biomedical engineering · 2024Article
- UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease.Blood advances · 2024Article
- CRISPR technology in human diseases.MedComm · 2024Review
- Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells.eLife · 2024Article
- Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models.Cytotherapy · 2024Article
- Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.Cell stem cell · 2024Article
- A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection.Cell stem cell · 2024Article
9 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 2 institutions in 1 country.
Funding
Abstract
Engineered nuclease-mediated gene targeting through homologous recombination (HR) in hematopoietic stem and progenitor cells (HSPCs) has the potential to treat a variety of genetic hematologic and immunologic disorders. Here, we identify critical parameters to reproducibly achieve high frequencies of RNA-guided (single-guide RNA [sgRNA]; CRISPR)-Cas9 nuclease (Cas9/sgRNA) and rAAV6-mediated HR at the β-globin (HBB) locus in HSPCs. We identified that by transducing HSPCs with rAAV6 post-electroporation, there was a greater than 2-fold electroporation-aided transduction (EAT) of rAAV6 endocytosis with roughly 70% of the cell population having undergone transduction within 2 hr. When HSPCs are cultured at low densities (1 × 10
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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.