International journal of biological macromoleculesHumansNanoparticlesCRISPR-Cas SystemsRetinoschisisRNA
Dual delivery of supramolecular nanoparticle-carried minicircle donor DNA with Cas9/gRNA improved HITI knock-in efficiency in X-linked juvenile retinoschisis.
Shih-Jie Chou, Chia-Hao Wang, Yuh-Lih Chang, Wei-Chieh Fang, De-Kuang Hwang, Yu-Jer Hsiao, Yung-Hung Luo, Wen-Liang Lo, Nguyen Quoc Viet, Kea-Yin Tang, Yuan-Tzu Lan, Chih-Chien Hsu, Shih-Jen Chen, Tai-Chi Lin, Yi-Ping Yang
Published: 202510.1016/j.ijbiomac.2025.148300
Abstract
X-linked retinoschisis (XLRS) is a hereditary mutation of the RS1 gene and is characterized by early-onset maculopathy with severe visual impairment. Current gene therapy utilizing CRISPR-associated protein 9 (Cas9) is ongoing; however, the optimizat…
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