Hiroyuki Nakai

The paper shows that AAV-mediated gene delivery to proximal tubules is not direct but relies on megalin-mediated uptake of reporter proteins from other organs, challenging current understanding.

The paper evaluates AAV capsids for liver gene therapy in a pig xenograft model, identifying efficient capsids for transgene expression in porcine hepatocytes.

The paper shows that GPR108 absence in birds explains lower AAV transduction efficacy and demonstrates that human GPR108 expression and capsid modifications enhance transduction in zebra finch cells.

This study shows that using a secretable TATk-CDKL5 protein in AAV gene therapy improves CDKL5 replacement and functional outcomes in Cdkl5 KO mice compared to native CDKL5 expression.

This study uses RNA base editing to repair a mutation in a mouse model of Rett syndrome, restoring MeCP2 function in hippocampal neurons.