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Yiling Long

Jinan University

Guangzhou

China

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Construction of a Self-Amplifying mRNA-LNP Complex for Phenylalanine Hydroxylase and Its Efficacy Evaluation in Gene Therapy for Phenylketonuria

Number of pages: 32 Posted: 23 Sep 2025
Ziqi Wen, Yiling Long, Rongjun Xu, Wenjing Shi, Xingri Zhan, Xiuyuan Wang, Rui Su, Jia Fei and Sneak Peek Administrator
Jinan University, Jinan University, Jinan University, Jinan University, Jinan University, Jinan University, Jinan University - Department of Medical Biochemistry and Molecular Biology, Jinan University - Department of Medical Biochemistry and Molecular Biology and Independent
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Abstract:

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phenylketonuria (PKU), phenylalanine hydroxylase (PAH), mRNA technology, self-amplifying mRNA, Lipid Nanoparticle (LNP), replacement therapy