Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases

por Luciani, Marco, Gritti, Angela, Meneghini, Vasco
Publicado 2020

In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges

por Cantore, Alessio, Fraldi, Alessandro, Meneghini, Vasco, Gritti, Angela
Publicado 2022

Editorial: Ex-vivo and in-vivo genome engineering for metabolic and neurometabolic diseases

por Colella, Pasqualina, Meneghini, Vasco, Baldo, Guilherme, Gomez-Ospina, Natalia
Publicado 2023

Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System

por Meneghini, Vasco, Peviani, Marco, Luciani, Marco, Zambonini, Giada, Gritti, Angela
Publicado 2021

GATA Factor-Mediated Gene Regulation in Human Erythropoiesis

por Romano, Oriana, Petiti, Luca, Felix, Tristan, Meneghini, Vasco, Portafax, Michel, Antoniani, Chiara, Amendola, Mario, Bicciato, Silvio, Peano, Clelia, Miccio, Annarita
Publicado 2020

Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy

por Frati, Giacomo, Luciani, Marco, Meneghini, Vasco, De Cicco, Silvia, Ståhlman, Marcus, Blomqvist, Maria, Grossi, Serena, Filocamo, Mirella, Morena, Francesco, Menegon, Andrea, Martino, Sabata, Gritti, Angela
Publicado 2018

Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

por Meneghini, Vasco, Frati, Giacomo, Sala, Davide, De Cicco, Silvia, Luciani, Marco, Cavazzin, Chiara, Paulis, Marianna, Mentzen, Wieslawa, Morena, Francesco, Giannelli, Serena, Sanvito, Francesca, Villa, Anna, Bulfone, Alessandro, Broccoli, Vania, Martino, Sabata, Gritti, Angela
Publicado 2016

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

por Weber, Leslie, Poletti, Valentina, Magrin, Elisa, Antoniani, Chiara, Martin, Samia, Bayard, Charles, Sadek, Hanem, Felix, Tristan, Meneghini, Vasco, Antoniou, Michael N., El-Nemer, Wassim, Mavilio, Fulvio, Cavazzana, Marina, Andre-Schmutz, Isabelle, Miccio, Annarita
Publicado 2018

Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy

por Meneghini, Vasco, Lattanzi, Annalisa, Tiradani, Luigi, Bravo, Gabriele, Morena, Francesco, Sanvito, Francesca, Calabria, Andrea, Bringas, John, Fisher‐Perkins, Jeanne M, Dufour, Jason P, Baker, Kate C, Doglioni, Claudio, Montini, Eugenio, Bunnell, Bruce A, Bankiewicz, Krystof, Martino, Sabata, Naldini, Luigi, Gritti, Angela
Publicado 2016

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

por Weber, Leslie, Frati, Giacomo, Felix, Tristan, Hardouin, Giulia, Casini, Antonio, Wollenschlaeger, Clara, Meneghini, Vasco, Masson, Cecile, De Cian, Anne, Chalumeau, Anne, Mavilio, Fulvio, Amendola, Mario, Andre-Schmutz, Isabelle, Cereseto, Anna, El Nemer, Wassim, Concordet, Jean-Paul, Giovannangeli, Carine, Cavazzana, Marina, Miccio, Annarita
Publicado 2020