Giulia Pavani

This paper introduces TILV, a CRISPR-assisted method for precise targeted integration of transgenes in human hematopoietic stem cells using IDLV donors, showing improved efficiency and minimal impact on cell viability.

This paper demonstrates that CRISPR-mediated transcriptional activation can upregulate SYNGAP1 in a mouse model of SRID and human neurons, rescuing deficits without targeting mutations.

This paper characterizes lysosomal storage disease using imaging and cytometry to assess lipid homeostasis in patient cells and validates findings in alternative models.

This paper identifies the NOD1-RIPK2-NFkB axis as a critical inflammatory pathway for hematopoietic stem cell specification from human pluripotent stem cells.