Open access
Sep 2026
R2Tg retrotransposon mediated targeted integration of exogenous genes into the 28S rDNA locus in mammalian cell lines for PKU gene therapy
Experimental results demonstrated that lipid nanoparticle (LNP) delivery of R2Tg facilitated efficient site-specific integration of EGFP and PAH transgenes in mammalian cells, significantly elevating PAH mRNA and protein expression without disrupting endogenous 28S rRNA levels.
Xiu-Yuan Wang, Zi-Qi Wen, Rong-Jun Xu et al.
· Frontiers in Genome Editing · 0 citations