Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in gene therapy using adeno-associated virus vectors (AAV), specifically “production of neutralizing antibodies” and “hepatotoxicity,” by employing a novel smart nanomachine equipped with AAV.

The research results are published in the journal ACS Nano.

The co-first authors of the paper are Assistant Professor Yuto Honda from the Laboratory for Chemistry and Life Science at the Institute of Science Tokyo. Dr. Hiroaki Kino, a Principal Research Scientist at iCONM, and Prof. Nishiyama are listed as corresponding authors alongside Prof. Honda, while other researchers from iCONM are acknowledged as co-authors.