Marcello Maresca

This study uses Prime Editors in zebrafish to achieve precise DNA insertion and substitution without exogenous donor DNA, showing higher efficiency for short modifications with nuclease-based PEn.

This paper describes a method to efficiently differentiate human iPSC into peptidergic nociceptors expressing CGRP and TRPV1, with functional maturity and drug response.

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.

The paper analyzes CRISPR-Cas9 off-target mutations and translocations at single-cell and tissue-specific levels in mice, revealing context-dependent variability in editing outcomes.

This paper demonstrates that replacing the BCL11A repressor binding site with a TAL1:GATA1 activator motif in HBG1/2 promoters enhances γ-globin reactivation and corrects sickle cell disease in hematopoietic stem/progenitor cells.

This study identifies Artemis as a regulator of PRINS editing, showing that modulating Artemis improves insertion efficiency and specificity for genomic engineering.

This paper describes a prime editing strategy to modify γ-globin promoters in HSPCs to increase HbF expression for sickle cell disease treatment.

This paper improves prime editing by modulating DNA repair pathways and engineering pegRNA, achieving higher precision and efficiency in human and mouse cells.

This study identifies AZD7648 and Pol{Theta} inhibitors as a combination (2iHDR) that enhances CRISPR-Cas9 integration efficiency and precision by targeting DNA-PK and MMEJ repair pathways.

This paper develops two prime editing strategies using DSB repair to improve genomic insertion efficiency and purity, expanding the prime editing toolkit.

This paper introduces an inducible CRISPR-Cas9 system for preclinical drug discovery, enabling controlled genome editing in mice to model cancer and test therapies.

This paper introduces DISCOVER-Seq, a method to detect CRISPR off-targets in vivo with single-base resolution, applicable to multiple Cas nucleases.

The paper introduces VIVO, a method to detect off-target mutations from in vivo CRISPR-Cas editing, showing that well-designed gRNAs can edit efficiently without detectable off-targets.