ArticleEBioMedicine2020
An effective vaginal gel to deliver CRISPR/Cas9 system encapsulated in poly (β-amino ester) nanoparticles for vaginal gene therapy.
Article in EBioMedicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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13 citing papers in PubMed, 30 citations in OpenAlex.
- Feasibility of combining JAK1 gene editing via CRISPR-CasRx with EGCG-lactoferrin nanoparticle therapy in a microneedle-based platform for atopic dermatitis.Materials today. Bio · 2026Article
- Unveiling the Potential of Nanoclays in Pharmaceuticals.AAPS PharmSciTech · 2025Review
- Mixed Infections in the Female Lower Genital Tract: Unlocking the Current Landscape and Future Directions.Current medical science · 2025Review
- A novel multilayer antimicrobial urinary catheter material with antimicrobial properties.Materials advances · 2025Article
- Advances in cervical cancer: current insights and future directions.Cancer communications (London, England) · 2025Review
- The Know-how of Polymeric Nanocarrier Based Vaginal Drug Delivery System: Pitfalls, Challenges and Trends.Pharmaceutical nanotechnology · 2025Review
- Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing.Small science · 2024Article
- Delivering CRISPR to the HIV-1 reservoirs.Frontiers in microbiology · 2024Review
- Poly(β-amino ester)s-based nanovehicles: Structural regulation and gene delivery.Molecular therapy. Nucleic acids · 2023Review
- Emerging Bioactive Agent Delivery-Based Regenerative Therapies for Lower Genitourinary Tissues.Pharmaceutics · 2022Review
- FrCas9 is a CRISPR/Cas9 system with high editing efficiency and fidelity.Nature communications · 2022Article
- Co-Delivery ofFrontiers in pharmacology · 2022Article
- The comparison of ZFNs, TALENs, and SpCas9 by GUIDE-seq in HPV-targeted gene therapy.Molecular therapy. Nucleic acids · 2021Article
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Authors and funding
33 authors at 13 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundGene therapy has held promises for treating specific genetic diseases. However, the key to clinical application depends on effective gene delivery.
methodsUsing a large animal model, we developed two pharmaceutical formulations for gene delivery in the pigs' vagina, which were made up of poly (β-amino ester) (PBAE)-plasmid polyplex nanoparticles (NPs) based two gel materials, modified montmorillonite (mMMT) and hectorite (HTT).
findingsBy conducting flow cytometry of the cervical cells, we found that PBAE-GFP-NPs-mMMT gel was more efficient than PBAE-GFP-NPs-HTT gel in delivering exogenous DNA intravaginally. Next, we designed specific CRISPR/SpCas9 sgRNAs targeting porcine endogenous retroviruses (PERVs) and evaluated the genome editing efficacy in vivo. We discovered that PERV copy number in vaginal epithelium could be significantly reduced by the local delivery of the PBAE-SpCas9/sgRNA NPs-mMMT gel. Comparable genome editing results were also obtained by high-fidelity version of SpCas9, SpCas9-HF1 and eSpCas9, in the mMMT gel. Further, we confirmed that the expression of topically delivered SpCas9 was limited to the vagina/cervix and did not diffuse to nearby organs, which was relatively safe with low toxicity.
interpretationOur data suggested that the PBAE-NPs mMMT vaginal gel is an effective preparation for local gene therapy, yielding insights into novel therapeutic approaches to sexually transmitted disease in the genital tract.
fundingThis work was supported by the National Science and Technology Major Project of the Ministry of science and technology of China (No. 2018ZX10301402); the National Natural Science Foundation of China (81761148025, 81871473 and 81402158); Guangzhou Science and Technology Programme (No. 201704020093); National Ten Thousand Plan-Young Top Talents of China, Fundamental Research Funds for the Central Universities (17ykzd15 and 19ykyjs07); Three Big Constructions-Supercomputing Application Cultivation Projects sponsored by National Supercomputer Center In Guangzhou; the National Research FFoundation (NRF) South Africa under BRICS Multilateral Joint Call for Proposals; grant 17-54-80078 from the Russian Foundation for Basic Research.
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