ArticleNature communications2018
CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration.
Article in Nature communications, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers.
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Who cites it
60 citing papers in PubMed, 98 citations in OpenAlex.
- Intravitreal Injection with a Conjunctival Injection Device: A Single-Center Experience.Translational vision science & technology · 2020Trial
- Advancements in CRISPR-based therapies for ocular pathologies: from disease mechanisms to intervention strategies.Theranostics · 2026Review
- The role of NLRP3 inflammasome in age-related macular degeneration: mechanisms and therapeutic prospects.Frontiers in aging neuroscience · 2026Review
- Engineered virus-like particle-assembled Vegfa-targeting Cas9 ribonucleoprotein treatment alleviates neovascularization in wet age-related macular degeneration.Genome biology · 2025Article
- Gene therapy in neovascular age related macular degeneration: an update.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2025Review
- Modular and signal-responsive transcriptional regulation using CRISPRi-aided genetic switches in Escherichia coli.Journal of biological engineering · 2025Article
- Exploitation of enhanced prime editing for blocking aberrant angiogenesis.Journal of advanced research · 2025Article
- Gene Therapy in Diabetic Retinopathy and Diabetic Macular Edema: An Update.Journal of clinical medicine · 2025Review
- Post-cleavage target residence determines asymmetry in non-homologous end joining of Cas12a-induced DNA double strand breaks.Genome biology · 2025Article
- Nanomaterial-Based Anti-Angiogenic Gene Therapy for Retinal Neovascular Diseases: Mechanistic Insights and Preclinical Advances.International journal of nanomedicine · 2025Review
- CRISPR-mediated ablation of TP53 and EGFR mutations enhances gefitinib sensitivity and anti-tumor efficacy in lung cancer.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Recent Insights into Roles of Hypoxia-Inducible Factors in Retinal Diseases.International journal of molecular sciences · 2024Review
- Multifunctional nano-in-micro delivery systems for targeted therapy in fundus neovascularization diseases.Journal of nanobiotechnology · 2024Review
- Gene targeting in adult organs using in vivo cleavable donor plasmids for CRISPR-Cas9 and CRISPR-Cas12a.Scientific reports · 2024Article
- A New Generation of Gene Therapies as the Future of Wet AMD Treatment.International journal of molecular sciences · 2024Review
- pH-Responsive polymer boosts cytosolic siRNA release for retinal neovascularization therapy.Acta pharmaceutica Sinica. B · 2024Article
- AAV-Based Strategies for Treatment of Retinal and Choroidal Vascular Diseases: Advances in Age-Related Macular Degeneration and Diabetic Retinopathy Therapies.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- Genome editing in the treatment of ocular diseases.Experimental & molecular medicine · 2023Review
- Long-Term Evaluation of Retinal Morphology and Function in Rosa26-Cas9 Knock-In Mice.International journal of molecular sciences · 2023Article
- CRISPR/Cas9 mediated specific ablation of vegfa in retinal pigment epithelium efficiently regresses choroidal neovascularization.Scientific reports · 2023Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
LbCpf1, derived from Lachnospiraceae bacterium ND2006, is a CRISPR RNA-guided endonuclease and holds promise for therapeutic applications. Here we show that LbCpf1 can be used for therapeutic gene editing in a mouse model of age-related macular degeneration (AMD). The intravitreal delivery of LbCpf1, targeted to two angiogenesis-associated genes encoding vascular endothelial growth factor A (Vegfa) and hypoxia inducing factor 1a (Hif1a), using adeno-associated virus, led to efficient gene disruption with no apparent off-target effects in the retina and retinal pigment epithelium (RPE) cells. Importantly, LbCpf1 targeted to Vegfa or Hif1a in RPE cells reduced the area of laser-induced choroidal neovascularization as efficiently as aflibercept, an anti-VEGF drug currently used in the clinic, without inducing cone dysfunction. Unlike aflibercept, LbCpf1 targeted to Vegfa or Hif1a achieved a long-term therapeutic effect on CNV, potentially avoiding repetitive injections. Taken together, these results indicate that LbCpf1-mediated in vivo genome editing to ablate pathologic angiogenesis provides an effective strategy for the treatment of AMD and other neovascularization-associated diseases.
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