ArticleMolecular therapy : the journal of the American Society of Gene Therapy2022
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway.
Article 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 32 papers.
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Who cites it
32 citing papers in PubMed, 47 citations in OpenAlex.
- Recombinant adeno-associated virus-mediated HSV-TK/GCV gene therapy for ovarian cancer: an experimental study.Translational cancer research · 2026Article
- Lung-targeted RNA delivery systems: strategies and therapeutic applications.Journal of nanobiotechnology · 2026Review
- Targeted delivery of genome editors in vivo.Nature biotechnology · 2026Review
- Sulfonium lipid nanoparticles for intranasal mRNA delivery to lung epithelial and immune cells.Acta biomaterialia · 2025Article
- Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing.Biomaterials · 2025Review
- Lung and liver editing by lipid nanoparticle delivery of a stable CRISPR-Cas9 ribonucleoprotein.Nature biotechnology · 2025Article
- Cross-species tropism of AAV.CPP.16 in the respiratory tract and its gene therapies against pulmonary fibrosis and viral infection.Cell reports. Medicine · 2025Article
- Article
- Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases.Current gene therapy · 2025Review
- CasRx-based Wnt activation promotes alveolar regeneration while ameliorating pulmonary fibrosis in a mouse model of lung injury.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys.Human gene therapy · 2024Article
- Nonviral CRISPR/Cas9 mutagenesis for streamlined generation of mouse lung cancer models.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets.International journal of molecular sciences · 2024Review
- Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy.Molecular cancer · 2024Review
- Article
- A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo.Nature communications · 2023Article
- Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X.Human molecular genetics · 2023Article
- Lipid nanoparticle-enabled gene editing in the lung via inhalation.Nature biotechnology · 2023Article
- Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing.Nature biotechnology · 2023Article
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Authors and funding
10 authors at 2 institutions in 1 country.
Funding
Abstract
Genome editing in the lung has the potential to provide long-term expression of therapeutic protein to treat lung genetic diseases. Yet efficient delivery of CRISPR to the lung remains a challenge. The NIH Somatic Cell Genome Editing (SCGE) Consortium is developing safe and effective methods for genome editing in disease tissues. Methods developed by consortium members are independently validated by the SCGE small animal testing center to establish rigor and reproducibility. We have developed and validated a dual adeno-associated virus (AAV) CRISPR platform that supports effective editing of a lox-stop-lox-Tomato reporter in mouse lung airway. After intratracheal injection of the AAV serotype 5 (AAV5)-packaged S. pyogenes Cas9 (SpCas9) and single guide RNAs (sgRNAs), we observed ∼19%-26% Tomato-positive cells in both large and small airways, including club and ciliated epithelial cell types. This highly effective AAV delivery platform will facilitate the study of therapeutic genome editing in the lung and other tissue types.
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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.