Brief intro:
- Author: Jiaxuan Jiang, Kangjie Kong, Xiuli Fang, Deming Wang, Yinhang Zhang, Peiyuan Wang, Zefeng Yang, Yuwei Zhang, Xiaoyi Liu, Tin Aung, Fei Li, Patrick Yu-Wai-Man, Xiulan Zhang
- Journal: Cell Reports Medicine
- Publication Date: 2024 May 21
Abstract
Highlights
- Carbonic anhydrase 2 (Car2) gene knockout can reduce intraocular pressure
- Dual-target CRISPR system mediates efficient knockout of Car2 gene in vivo
- A single intravitreal injection can maintain long-term IOP reduction
- CRISPR-Cas9-mediated Car2 gene knockout as a gene therapy for glaucoma
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PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
