iScience· 2026Q1

Construction of a self-amplifying mRNA-LNP complex for phenylalanine hydroxylase and its efficacy evaluation in gene therapy for phenylketonuria

Ziqi Wen, Yiling Long, Wenjing Shi, Wenjing Song et al.

0citations
Q1SCImago
2026year

Short summary

A self-amplifying mRNA (saRNA) delivered via lipid nanoparticles (LNPs) achieved sustained phenylalanine hydroxylase (PAH) activity for 14 days in mice with phenylketonuria (PKU), requiring one-sixth the dose of conventional mRNA and showing no toxicity.

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Clinical BiochemistryBiochemistry, Genetics and Molecular Biology