Luca Biasco

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

This paper develops a statistical model to infer hematopoietic stem cell dynamics in a gene therapy trial for Wiskott-Aldrich Syndrome and finds evidence for a myeloid-based developmental pathway.

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.

CRISPR-SURF deconvolves CRISPR tiling screen data to identify functional regulatory elements across CRISPR-Cas, CRISPRi, and CRISPRa experiments.