ArticleScience advances2021
Genome-editing prodrug: Targeted delivery and conditional stabilization of CRISPR-Cas9 for precision therapy of inflammatory disease.
Article in Science advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers.
What it found
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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
37 citing papers in PubMed, 82 citations in OpenAlex.
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Advances in immune cell-based therapeutic agents for the treatment of inflammation-related diseases.Acta pharmaceutica Sinica. B · 2026Review
- 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
- Nature Inspired Delivery Vehicles for CRISPR-Based Genome Editing.Small (Weinheim an der Bergstrasse, Germany) · 2026Review
- In vivo chemogenetic RNA editing of macrophages by bioengineered viruses for sepsis treatment.Nature communications · 2025Article
- Reshaping tumor immune microenvironment through ROS-responsive prodrug polyplexes via synergistic effect of CRISPRi system and epigenetic inhibitor for breast cancer therapy.Materials today. Bio · 2025Article
- Polydopamine Nanodots Ameliorate Inflammatory Bowel Disease by Restoring Redox Homeostasis and Intestinal Microenvironment.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Engineered plant extracellular vesicles: Emerging nanoplatforms for combinational cancer immunotherapy.Acta pharmaceutica Sinica. B · 2025Review
- Review
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- Nanoscale synthetic biology with innovative medicinal applications.Fundamental research · 2025Review
- Application of single-cell sequencing in the study of immune cell infiltration in inflammatory bowel disease and colorectal cancer.World journal of gastrointestinal oncology · 2025Review
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- Reactive oxygen species responsive nanomotors for gene edited metabolic disruption and immunotherapy.Nature communications · 2025Article
- Advancing cancer gene therapy: the emerging role of nanoparticle delivery systems.Journal of nanobiotechnology · 2025Review
- Bionic gene delivery system activates tumor autophagy and immunosuppressive niche to sensitize anti-PD-1 treatment against STK11-mutated lung adenocarcinoma.Journal of nanobiotechnology · 2025Article
- Inhalable liposomal delivery of osimertinib and DNA for treating primary and metastasis lung cancer.Nature communications · 2025Article
- Prime Editing: A Revolutionary Technology for Precise Treatment of Genetic Disorders.Cell proliferation · 2025Review
- Finding a needle in a haystack: functional screening for novel targets in cancer immunology and immunotherapies.Oncogene · 2025Review
- CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations.Molecules (Basel, Switzerland) · 2025Review
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Regulation of CRISPR-Cas9 functions in vivo is conducive to developing precise therapeutic genome editing. Here, we report a CRISPR-Cas9 prodrug nanosystem (termed NanoProCas9), which combines the targeted delivery and the conditional activation of CRISPR-Cas9 for the precision therapy of inflammatory bowel disease. NanoProCas9 is composed of (i) cationic poly(β-amino ester) (PBAE) capable of complexing plasmid DNA encoding destabilized Cas9 (dsCas9) nuclease, (ii) a layer of biomimetic cell membrane coated on PBAE/plasmid nanocomplexes for the targeted delivery of PBAE/dsCas9 complexes, and (iii) the stimuli-responsive precursory molecules anchored on the exofacial membrane. The systemic administration of NanoProCas9 enables the targeted delivery of dsCas9 plasmid into inflammatory lesions, where the precursory small molecule can be activated by ROS signals to stabilize expressed dsCas9, thereby activating Cas9 function for inflammatory genome editing. The proposed “genome-editing prodrug” presents a proof-of-concept example to precisely regulate CRISPR-Cas9 functions by virtue of particular pathological stimuli in vivo.
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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.