Édina Poletto

In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system

Roselena Silvestri Schuh, Édina Poletto, Gabriela Pasqualim et al. · Journal of Controlled Release · 2018 · 68 citations

Knockout Mouse, Off-target Effect, Engineering +13

Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

Samantha G. Scharenberg, Édina Poletto, Katherine L. Lucot et al. · Nature Communications · 2020 · 55 citations · Full text

Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Roselena Silvestri Schuh, Esteban Alberto Gonzalez, Ângela Maria Vicente Tavares et al. · Gene Therapy · 2019 · 34 citations · Full text

Knockout Mouse, Gene Therapies, Bone Disease Features +12

Cathepsin B inhibition attenuates cardiovascular pathology in mucopolysaccharidosis I mice

Esteban Alberto Gonzalez, Giselle Renata Martins, Ângela Maria Vicente Tavares et al. · Life Sciences · 2018 · 30 citations

Inflammation, Disease Pathophysiology, Medicine +6

Nasal Administration of Cationic Nanoemulsions as Nucleic Acids Delivery Systems Aiming at Mucopolysaccharidosis Type I Gene Therapy

Roselena Silvestri Schuh, Juliana Bidone, Édina Poletto et al. · Pharmaceutical Research · 2018 · 28 citations

Cell-based Drug Delivery, Nanomedicine, Gene Therapies +9