Giuseppe Ronzitti

The paper describes the development of En-ISPmu1, a modified TnpB nuclease, and demonstrates its ability to achieve targeted genome integration in mouse liver with a single AAV vector.

The paper shows that immune responses to AAV-mediated ocular gene delivery vary across species and individuals, complicating reliable immunomonitoring.

The paper develops a liver-targeted gene therapy using recombinant AAV8 to cure Lysosomal acid lipase deficiency by correcting lipid accumulation and normalizing gene expression and mitochondrial function in a mouse model.

This study uses CRISPR-Cas9 to disrupt miR binding sites and upregulate utrophin in DMD models, showing functional improvements and therapeutic potential.