ArticleMolecular therapy. Nucleic acids2020
Poly(Beta-Amino Ester) Nanoparticles Enable Nonviral Delivery of CRISPR-Cas9 Plasmids for Gene Knockout and Gene Deletion.
Article in Molecular therapy. Nucleic acids, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.
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Who cites it
34 citing papers in PubMed, 68 citations in OpenAlex.
- Non-Viral CRISPR carriers: transient delivery with lasting effects.Drug delivery · 2026Review
- Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis.JCI insight · 2026Article
- Nanomaterials in gene therapy and genome editing: challenges and emerging directions.Journal of nanobiotechnology · 2025Review
- Base editing and nanoparticle transfection of airway cell types essential for treatment of cystic fibrosis.bioRxiv : the preprint server for biology · 2025Article
- In vivo CAR engineering for immunotherapy.Nature reviews. Immunology · 2025Review
- Glutathione hybrid poly (beta-amino ester)-plasmid nanoparticles for enhancing gene delivery and biosafety.Journal of advanced research · 2025Article
- CRISPR-Cas systems against carbapenem resistance: from proof-of-concept to clinical translation.Frontiers in microbiology · 2025Review
- Advances and prospects of RNA delivery nanoplatforms for cancer therapy.Acta pharmaceutica Sinica. B · 2025Review
- Nucleic acid-loaded poly(beta-aminoester) nanoparticles for cancer nano-immuno therapeutics: the good, the bad, and the future.Drug delivery and translational research · 2024Review
- Current Knowledge on CRISPR Strategies Against Antimicrobial-Resistant Bacteria.Antibiotics (Basel, Switzerland) · 2024Review
- Unlocking Genome Editing: Advances and Obstacles in CRISPR/Cas Delivery Technologies.Journal of functional biomaterials · 2024Review
- Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing.Small science · 2024Article
- Role of CRISPR-Cas systems and anti-CRISPR proteins in bacterial antibiotic resistance.Heliyon · 2024Review
- Elucidating the Effect of Amine Charge State on Poly(β-amino ester) Degradation Using Permanently Charged Analogs.ACS macro letters · 2023Article
- CRISPR-Cas-Based Antimicrobials: Design, Challenges, and Bacterial Mechanisms of Resistance.ACS infectious diseases · 2023Review
- The changing landscape of agriculture: role of precision breeding in developing smart crops.Functional & integrative genomics · 2023Review
- BSA-PEI Nanoparticle Mediated Efficient Delivery of CRISPR/Cas9 into MDA-MB-231 Cells.Molecular biotechnology · 2022Article
- Approaches towards biomaterial-mediated gene editing for cancer immunotherapy.Biomaterials science · 2022Review
- Research Progress on Nanoparticles-Based CRISPR/Cas9 System for Targeted Therapy of Tumors.Biomolecules · 2022Review
- Delivery of Therapeutic miR-148b Mimic via Poly(β Amino Ester) Polyplexes for Post-transcriptional Gene Regulation and Apoptosis of A549 Cells.Langmuir : the ACS journal of surfaces and colloids · 2022Article
Corrections and comments
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
The CRISPR-Cas9 system is a powerful gene-editing tool with wide-ranging applications, but the safe and efficient intracellular delivery of CRISPR components remains a challenge. In this study, we utilized biodegradable poly(beta-amino ester) nanoparticles to codeliver plasmid DNA encoding Cas9 and short guide RNA (sgRNA), respectively, to enable gene knockout following a CRISPR-mediated cleavage at one genomic site (1-cut edit), as well as gene deletion following DNA cleavage at two sites flanking a region of interest (2-cut edits). We designed a reporter system that allows for easy evaluation of both types of edits: gene knockout can be assessed by a decrease in near-infrared fluorescent protein (iRFP) fluorescence, whereas deletion of an expression stop cassette turns on a red-enhanced nanolantern fluorescence/luminescence dual reporter. Nanoparticles enabled up to 70% gene knockout due to small indels, as well as 45% gain-of-function expression after a 600-bp deletion edit. The efficiency of 2-cut edits is more sensitive than 1-cut edits to Cas9 and the sgRNA expression level. We demonstrate promising biodegradable nanoparticle formulations for gene editing. Our findings also provide new insights into the screening and transfection requirements for different types of gene edits, which are applicable for designing nonviral delivery systems for the CRISPR-Cas9 platform.
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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.