Cícera Regina Lazzarotto

This paper describes a gene-engineered B Cell Medicine (BE-101) that produces sustained active factor IX for hemophilia B therapy, showing durable and redosable effects in mice.

The paper uses population-scale GUIDE-seq-2 and CHANCE-seq to analyze how genetic variants affect Cas9 activity, finding frequent context-dependent effects on off-target sites.

This study develops a genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease, showing efficient editing, therapeutic HbF levels, and no off-target effects.

The paper introduces CHANGE-seq-BE, a method for sensitive and unbiased profiling of base editor off-target effects in vitro.

This paper describes the optimization of base editing to correct the SMN2 exon 7 mutation in SMA, achieving high editing efficiency and increased SMN protein levels in vitro and in vivo.

This paper compares CRISPR-mediated HDR and PE2 systems in mice to show PE2's higher fidelity in editing a TFBS, leading to more effective tissue-specific gene inactivation.

The paper validates off-target effects of CRISPR/Cas9 in rhesus macaques using error-corrected sequencing, showing that in vitro and in silico methods have limited predictive accuracy.