Alessio Cremonesi

This study uses isogenic brain organoids to identify early neurodevelopmental changes and hyperexcitability in Gaucher disease, linking lipid dysregulation and neuronal function imbalances to clinical symptoms.

This paper demonstrates transient prime editor expression in mice to correct a genetic liver disease, reducing blood phenylalanine levels below the therapeutic threshold.

This study uses base editing to downregulate PTBP1 in neurons of a PD mouse model, rescuing motor symptoms without affecting astrocytes.

This study shows that GLUD1 controls muscle stem cell differentiation by regulating mitochondrial glutamate levels and the malate-aspartate shuttle.