ArticleNature materials2019
Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations.
Article in Nature materials, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 84 papers, 1 of them a synthesis that pooled it.
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
84 citing papers in PubMed, 1 synthesis or guideline pooled it.
- CRISPR-Cas systems in combating antimicrobial resistance: which system to choose? A systematic review.World journal of microbiology & biotechnology · 2025Pooled it
- Non-Viral CRISPR carriers: transient delivery with lasting effects.Drug delivery · 2026Review
- Nanoengineering Systems for Gene Therapy: Mechanisms, Modalities, and Future Directions.International journal of molecular sciences · 2026Review
- AI-nanotech synergies: recent advances in sustainable bio-manufacturing.World journal of microbiology & biotechnology · 2026Review
- In vivo production of an anti-HIV antibody in mice by non-viral gene knockin in primate hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Nonviral Gold Nanoparticle-Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes Into Primary Blood Cells.Advanced nanobiomed research · 2026Article
- CRISPR-AuNP: physicochemical optimization of a gold nanoparticle platform for cost-effective and modular non-viral gene editing in HSPCs.Gene therapy · 2026Article
- Anatomically Guided Non-Viral CRISPR/Cas9 Delivery in the Eye: Overcoming Barriers for Precision Gene Therapy.Pharmaceutics · 2026Review
- Nanocarrier-mediated CRISPR-Cas delivery: a novel approach against antibiotic-resistant superbugs.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2026Review
- Gold Nanoparticles in Biomedical Applications: Synthesis, Functionalization, and Recent Advances.Molecules (Basel, Switzerland) · 2025Review
- Nanomaterials in gene therapy and genome editing: challenges and emerging directions.Journal of nanobiotechnology · 2025Review
- Enhanced hematopoietic stem cell self-renewal and engraftment through human lung exosomal communication.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- A general genome editing strategy using CRISPR lipid nanoparticle spherical nucleic acids.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Harnessing an integrated glyco-nanovaccine technology for enhanced cancer immunotherapy.Communications medicine · 2025Article
- Innovative approaches to combat antibiotic resistance: integrating CRISPR/Cas9 and nanoparticles against biofilm-driven infections.BMC medicine · 2025Review
- CRISPR-Cas technologies: Emerging tools from research to clinical application.Journal of microbiology (Seoul, Korea) · 2025Review
- Identifying optimal conditions for precise knock-in of exogenous DNA into the zebrafish genome.Development (Cambridge, England) · 2025Article
- Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.Molecular therapy. Nucleic acids · 2025Review
- SynBioNanoDesign: pioneering targeted drug delivery with engineered nanomaterials.Journal of nanobiotechnology · 2025Review
- Excision of HIV-1 Provirus in Human Primary Cells with Nanocapsuled TALEN Proteins.ACS applied bio materials · 2025Article
24 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
8 authors.
Funding
Abstract
Ex vivo CRISPR gene editing in haematopoietic stem and progenitor cells has opened potential treatment modalities for numerous diseases. The current process uses electroporation, sometimes followed by virus transduction. While this complex manipulation has resulted in high levels of gene editing at some genetic loci, cellular toxicity was observed. We have developed a CRISPR nanoformulation based on colloidal gold nanoparticles with a unique loading design capable of cellular entry without the need for electroporation or viruses. This highly monodispersed nanoformulation avoids lysosomal entrapment and localizes to the nucleus in primary human blood progenitors without toxicity. Nanoformulation-mediated gene editing is efficient and sustained with different CRISPR nucleases at multiple loci of therapeutic interest. The engraftment kinetics of nanoformulation-treated primary cells in humanized mice are better relative to those of non-treated cells, with no differences in differentiation. Here we demonstrate non-toxic delivery of the entire CRISPR payload into primary human blood progenitors.
Indexed as
Identifiers
What OpenQuestion holds
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.