Mariana Loperfido

This study shows lentiviral HSPC gene therapy can reverse Pompe disease symptoms in mice by correcting muscle and neurological issues.

IS-Seq is a bioinformatics pipeline that integrates UMI-based and other methods for IS abundance estimation, showing UMI provides better accuracy than sonication fragment counts in clinical scenarios.

This study shows genetically engineered microglia-like cells can provide therapeutic protein levels in mouse models of neurodegenerative diseases.