ReviewMolecular therapy : the journal of the American Society of Gene Therapy2022
New approaches to moderate CRISPR-Cas9 activity: Addressing issues of cellular uptake and endosomal escape.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed, 32 citations in OpenAlex.
- Cell-Penetrating Peptide Delivery for CRISPR Applications.Methods in molecular biology (Clifton, N.J.) · 2027Article
- CRISPR-Cas9 in Cancer Therapy: Overcoming Delivery Barriers and Off-Target Effects for Clinical Translation.Health science reports · 2026Article
- Research Advances on Organelle-Targeted Drug Delivery Systems for the Treatment of Brain Tumors and Central Nervous System Inflammation.Pharmaceutics · 2026Review
- Precision Lipid Management in the Era of Biotechnology and Artificial Intelligence: From Gene Editing to Smart Drug Delivery.Therapeutic innovation & regulatory science · 2026Review
- Nanoengineering Systems for Gene Therapy: Mechanisms, Modalities, and Future Directions.International journal of molecular sciences · 2026Review
- Organoid-based systems for biomedical innovation: advances in disease modeling, drug screening, and precision medicine.Naunyn-Schmiedeberg's archives of pharmacology · 2026Review
- Nanomaterials in gene therapy and genome editing: challenges and emerging directions.Journal of nanobiotechnology · 2025Review
- A multistep platform identifies spleen-tropic lipid nanoparticles for in vivo T cell-targeted delivery of gene-editing proteins.Science advances · 2025Article
- Multifunctional gold nanoparticles: bridging detection, diagnosis, and targeted therapy in cancer.Molecular cancer · 2025Review
- Endo/Lysosomal-Escapable Lipid Nanoparticle Platforms for Enhancing mRNA Delivery in Cancer Therapy.Pharmaceutics · 2025Review
- Towards the elimination of infectious HPV: exploiting CRISPR/Cas innovations.Frontiers in cellular and infection microbiology · 2025Review
- Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.Molecular therapy. Nucleic acids · 2023Review
- CRISPR/Cas9 system: recent applications in immuno-oncology and cancer immunotherapy.Experimental hematology & oncology · 2023Review
- CRISPR/dCas9 for hepatic fibrosis therapy: implications and challenges.Molecular biology reports · 2022Review
- Improving Stability and Specificity of CRISPR/Cas9 System by Selective Modification of Guide RNAs with 2'-fluoro and Locked Nucleic Acid Nucleotides.International journal of molecular sciences · 2022Article
- In vitro reconstitution of calcium-dependent recruitment of the human ESCRT machinery in lysosomal membrane repair.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- Multidrug-Resistant Microbial Therapy Using Antimicrobial Peptides and the CRISPR/Cas9 System.Veterinary medicine (Auckland, N.Z.) · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
CRISPR-Cas9 is rapidly entering molecular biology and biomedicine as a promising gene-editing tool. A unique feature of CRISPR-Cas9 is a single-guide RNA directing a Cas9 nuclease toward its genomic target. Herein, we highlight new approaches for improving cellular uptake and endosomal escape of CRISPR-Cas9. As opposed to other recently published works, this review is focused on non-viral carriers as a means to facilitate the cellular uptake of CRISPR-Cas9 through endocytosis. The majority of non-viral carriers, such as gold nanoparticles, polymer nanoparticles, lipid nanoparticles, and nanoscale zeolitic imidazole frameworks, is developed with a focus toward optimizing the endosomal escape of CRISPR-Cas9 by taking advantage of the acidic environment in the late endosomes. Among the most broadly used methods for in vitro and ex vivo ribonucleotide protein transfection are electroporation and microinjection. Thus, other delivery formats are warranted for in vivo delivery of CRISPR-Cas9. Herein, we specifically revise the use of peptide and nanoparticle-based systems as platforms for CRISPR-Cas9 delivery in vivo. Finally, we highlight future perspectives of the CRISPR-Cas9 gene-editing tool and the prospects of using non-viral vectors to improve its bioavailability and therapeutic potential.
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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.