Emerging Issues in AAV-Mediated In Vivo Gene Therapy

por Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Publicado 2017

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

Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration

por Colella, Pasqualina, Iodice, Carolina, Di Vicino, Umberto, Annunziata, Ida, Surace, Enrico M., Auricchio, Alberto
Publicado 2011

Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation

por Poletto, Edina, Colella, Pasqualina, Pimentel Vera, Luisa N., Khan, Shaukat, Tomatsu, Shunji, Baldo, Guilherme, Gomez-Ospina, Natalia
Publicado 2022

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

por Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico
Publicado 2016

Myosin7a Deficiency Results in Reduced Retinal Activity Which Is Improved by Gene Therapy

por Colella, Pasqualina, Sommella, Andrea, Marrocco, Elena, Di Vicino, Umberto, Polishchuk, Elena, Garrido, Marina Garcia, Seeliger, Mathias W., Polishchuk, Roman, Auricchio, Alberto
Publicado 2013

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

por Scharenberg, Samantha G., Poletto, Edina, Lucot, Katherine L., Colella, Pasqualina, Sheikali, Adam, Montine, Thomas J., Porteus, Matthew H., Gomez-Ospina, Natalia
Publicado 2020

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

por Scharenberg, Samantha G., Poletto, Edina, Lucot, Katherine L., Colella, Pasqualina, Sheikali, Adam, Montine, Thomas J., Porteus, Matthew H., Gomez-Ospina, Natalia
Publicado 2020

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle

por Poupiot, Jérôme, Costa Verdera, Helena, Hardet, Romain, Colella, Pasqualina, Collaud, Fanny, Bartolo, Laurent, Davoust, Jean, Sanatine, Peggy, Mingozzi, Federico, Richard, Isabelle, Ronzitti, Giuseppe
Publicado 2019

Effective delivery of large genes to the retina by dual AAV vectors

por Trapani, Ivana, Colella, Pasqualina, Sommella, Andrea, Iodice, Carolina, Cesi, Giulia, de Simone, Sonia, Marrocco, Elena, Rossi, Settimio, Giunti, Massimo, Palfi, Arpad, Farrar, Gwyneth J, Polishchuk, Roman, Auricchio, Alberto
Publicado 2014

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

por Trapani, Ivana, Toriello, Elisabetta, de Simone, Sonia, Colella, Pasqualina, Iodice, Carolina, Polishchuk, Elena V., Sommella, Andrea, Colecchi, Linda, Rossi, Settimio, Simonelli, Francesca, Giunti, Massimo, Bacci, Maria L., Polishchuk, Roman S., Auricchio, Alberto
Publicado 2015

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

por Colella, Pasqualina, Sellier, Pauline, Costa Verdera, Helena, Puzzo, Francesco, van Wittenberghe, Laetitia, Guerchet, Nicolas, Daniele, Nathalie, Gjata, Bernard, Marmier, Solenne, Charles, Severine, Simon Sola, Marcelo, Ragone, Isabella, Leborgne, Christian, Collaud, Fanny, Mingozzi, Federico
Publicado 2018

CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency

por Colella, Pasqualina, Sayana, Ruhi, Suarez-Nieto, Maria Valentina, Sarno, Jolanda, Nyame, Kwamina, Xiong, Jian, Vera, Luisa Natalia Pimentel, Basurto, Jessica Arozqueta, Corbo, Marco, Limaye, Anay, Davis, Kara Lynn, Abu-Remaileh, Monther, Gomez-Ospina, Natalia
Publicado 2023

Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

por Pontoizeau, Clément, Simon-Sola, Marcelo, Gaborit, Clovis, Nguyen, Vincent, Rotaru, Irina, Tual, Nolan, Colella, Pasqualina, Girard, Muriel, Biferi, Maria-Grazia, Arnoux, Jean-Baptiste, Rötig, Agnès, Ottolenghi, Chris, de Lonlay, Pascale, Mingozzi, Federico, Cavazzana, Marina, Schiff, Manuel
Publicado 2022

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

por Masat, Elisa, Laforêt, Pascal, De Antonio, Marie, Corre, Guillaume, Perniconi, Barbara, Taouagh, Nadjib, Mariampillai, Kuberaka, Amelin, Damien, Mauhin, Wladimir, Hogrel, Jean-Yves, Caillaud, Catherine, Ronzitti, Giuseppe, Puzzo, Francesco, Kuranda, Klaudia, Colella, Pasqualina, Mallone, Roberto, Benveniste, Olivier, Mingozzi, Federico
Publicado 2016

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

por Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, Van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Collaud, Fanny, Charles, Severine, Sola, Marcelo Simon, Boyer, Olivier, Krijnse-Locker, Jacomina, Ronzitti, Giuseppe, Colella, Pasqualina, Mingozzi, Federico
Publicado 2020

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

por Colella, Pasqualina, Sellier, Pauline, Gomez, Manuel J., Biferi, Maria G., Tanniou, Guillaume, Guerchet, Nicolas, Cohen-Tannoudji, Mathilde, Moya-Nilges, Maryse, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Krijnse-Locker, Jacomina, Collaud, Fanny, Simon-Sola, Marcelo, Charles, Severine, Cagin, Umut, Mingozzi, Federico
Publicado 2020

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

por Costa-Verdera, Helena, Collaud, Fanny, Riling, Christopher R., Sellier, Pauline, Nordin, Jayme M. L., Preston, G. Michael, Cagin, Umut, Fabregue, Julien, Barral, Simon, Moya-Nilges, Maryse, Krijnse-Locker, Jacomina, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Cosette, Jeremie, Abad, Catalina, Simon-Sola, Marcelo, Charles, Severine, Li, Mathew, Crosariol, Marco, Antrilli, Tom, Quinn, William J., Gross, David A., Boyer, Olivier, Anguela, Xavier M., Armour, Sean M., Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Publicado 2021