Engineered virus-like particle-assembled Vegfa-targeting Cas9 ribonucleoprotein treatment alleviates neovascularization in wet age-related macular degeneration
eVLPs exhibit the potential as a safe and efficient delivery platform for Cas9 ribonucleoproteins, achieving precise Vegfa downregulation and significant reduction in choroidal neovascularization in a mouse model of wet age-related macular degeneration. With transient delivery of gene editing enzymes, high editing efficiency, and minimal risk of genomic integration, eVLPs present a promising alternative to conventional delivery systems for advancing genome editing therapies in retinal diseases.
Authors
- Maren van Son (ORCID: https://orcid.org/0000-0002-4892-3387)
- Hyewon Jang
- Hyunjong Kwak
- Jeong Hun Kim (ORCID: https://orcid.org/0000-0003-2957-1766)
- Seokjoong Kim (ORCID: https://orcid.org/0000-0002-4693-738X)
- Wei-Yan Jiang
- Hye‐Yeon Hwang (ORCID: https://orcid.org/0000-0002-7918-728X)
- Daesik Kim (ORCID: https://orcid.org/0000-0003-0806-5979)
- Dong Hyun Jo (ORCID: https://orcid.org/0000-0002-6320-6829)
- Jun Wu (ORCID: https://orcid.org/0000-0003-0807-5229)
Institutions
- Seoul National University (KR)
- Yonsei University (KR)
- Seoul National University Hospital (KR)
- National University (SD)
- Institute for Basic Science (KR)
- National University College (PR)
- Yonsei University Health System (KR)
- Sungkyunkwan University (KR)
Publication Details
- Journal
- Genome biology
- Published
- 2025-10-09
- DOI
- https://doi.org/10.1186/s13059-025-03774-5
- Citations
- 1
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.81
Funders
- National Research Foundation
- Seoul National University
- National Research Foundation of Korea
- Seoul National University Hospital
- Ministry of Science and ICT, South Korea