ArticleIntegrative biology : quantitative biosciences from nano to macro2017
Highly efficient genome editing of human hematopoietic stem cells via a nano-silicon-blade delivery approach.
Article in Integrative biology : quantitative biosciences from nano to macro, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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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.
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Who cites it
16 citing papers in PubMed, 34 citations in OpenAlex.
- Advances in nanomaterial-mediated CRISPR/Cas delivery: from lipid nanoparticles to vesicle-derived systems.Frontiers in bioengineering and biotechnology · 2025Review
- Exploring non-viral methods for the delivery of CRISPR-Cas ribonucleoprotein to hematopoietic stem cells.Stem cell research & therapy · 2024Review
- Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches.International journal of molecular sciences · 2024Review
- Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells.Stem cell reviews and reports · 2023Review
- Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells.ACS applied materials & interfaces · 2023Article
- CRISPR medicine for blood disorders: Progress and challenges in delivery.Frontiers in genome editing · 2022Review
- Microfluidic and Nanofluidic Intracellular Delivery.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Review
- Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing.Theranostics · 2021Review
- Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing.Pharmaceutics · 2020Review
- Lipid-Bicelle-Coated Microfluidics for Intracellular Delivery with Reduced Fouling.ACS applied materials & interfaces · 2020Article
- Improved Isolation and Culture of Urine-Derived Stem Cells (USCs) and Enhanced Production of Immune Cells from the USC-Derived Induced Pluripotent Stem Cells.Journal of clinical medicine · 2020Article
- 3,2'-Dihydroxyflavone Improves the Proliferation and Survival of Human Pluripotent Stem Cells and Their Differentiation into Hematopoietic Progenitor Cells.Journal of clinical medicine · 2020Article
- A review of emerging physical transfection methods for CRISPR/Cas9-mediated gene editing.Theranostics · 2020Review
- Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.PloS one · 2019Article
- TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells.Scientific reports · 2018Article
- Intracellular Delivery by Membrane Disruption: Mechanisms, Strategies, and Concepts.Chemical reviews · 2018Review
Corrections and comments
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Authors and funding
13 authors at 4 institutions in 5 countries.
Funding
Abstract
Recently, the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 bacterial immunity system has opened a promising avenue to treat genetic diseases that affect the human hematopoietic stem cells (HSCs). Therefore, finding a highly efficient delivery method capable of modifying the genome in the hard-to-transfect HSCs, combined with the advanced CRISPR-Cas9 system, may meet the challenges for dissecting the hematologic disease mechanisms and facilitate future clinical applications. Here, we developed an effective HSC-specified delivery microfluidic chip to disrupt the cell membrane transiently by inducing rapid mechanical deformation that allowed the delivery of biomaterials into the cytoplasm from the surrounding matrix. Compared with the previous designs, the new nano-silicon-blade structure was specifically optimized for HSCs. Using the silicon substrate, the sharpness and rigidity of the nano-blade constriction was largely enhanced to improve the biomaterials delivery efficiency. We achieved highly efficient delivery results by transporting various macro-molecules into the HSCs. Moreover, the treated HSCs possess high viability and maintain inherent pluripotency after the delivery via the Nano-Blade Chip (NB-Chip). Subsequently, we disrupted the p42 isoform in C/EBPα on the NB-Chip and induced HSCs into a myeloid proliferation behavior. In conclusion, the NB-Chip provides a harmless, rapid and high-throughput gene editing approach for the HSC study and therapeutics.
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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.