P. Colella

发表

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

G. Ronzitti, Laetitia van Wittenberghe, F. Mingozzi, 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

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

Laetitia van Wittenberghe, F. Mingozzi, B. Gjata, 2020, EBioMedicine.

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

X. Anguela, G. Ronzitti, Laetitia van Wittenberghe, 2021, Nature Communications.

Gene therapy for Pompe disease: the time is now.

F. Mingozzi, P. Colella, 2019, Human gene therapy.

AP20187-mediated activation of a chimeric insulin receptor results in insulin-like actions in skeletal muscle and liver of diabetic mice.

P. Formisano, F. Beguinot, Ferdinando Giacco, 2007, Human gene therapy.

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

F. Chapon, G. Bassez, D. Orlikowski, 2016, Scientific Reports.

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

Laetitia van Wittenberghe, C. Leborgne, F. Mingozzi, 2018, Molecular therapy. Methods & clinical development.

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

N. Gomez-Ospina, S. Tomatsu, E. Poletto, 2022, Molecular therapy. Methods & clinical development.

Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity.

J. Davoust, J. Demengeot, G. Ronzitti, 2019, JCI insight.

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

M. Seeliger, R. Polishchuk, A. Auricchio, 2013, PloS one.

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

T. Montine, M. Porteus, N. Gomez-Ospina, 2020, Nature Communications.

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

A. Rötig, P. de Lonlay, F. Mingozzi, 2022, Nature Communications.

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

G. Comi, E. Malfatti, I. Richard, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

M. Bacci, A. Auricchio, S. Rossi, 2014, Gene Therapy.

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

G. Ronzitti, F. Mingozzi, Federico Mingozzi, 2017, Molecular therapy. Methods & clinical development.

Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.

A. Auricchio, P. Colella, 2012, Human gene therapy.

TFEB Links Autophagy to Lysosomal Biogenesis

Andrea Ballabio, A. Ballabio, S. Erdin, 2011, Science.

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

M. Pinotti, G. Ronzitti, Laetitia van Wittenberghe, 2016, Molecular therapy. Nucleic acids.

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

R. Polishchuk, A. Auricchio, S. Rossi, 2013, EMBO molecular medicine.

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

M. Bacci, R. Polishchuk, A. Auricchio, 2015, Human molecular genetics.

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

J. Bennett, David S. Williams, S. Kim, 2008, The Journal of clinical investigation.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

M. Kay, P. Laforêt, G. Ronzitti, 2017, Science Translational Medicine.

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

T. Montine, M. Porteus, N. Gomez-Ospina, 2020, Nature Communications.

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

E. Surace, Ida Annunziata, A. Auricchio, 2011, Human molecular genetics.

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

J. Davoust, I. Richard, G. Ronzitti, 2019, Molecular therapy. Methods & clinical development.

Ocular gene therapy: current progress and future prospects.

A. Auricchio, G. Cotugno, P. Colella, 2009, Trends in molecular medicine.

AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases.

A. Auricchio, P. Colella, 2010, Current gene therapy.

Photoreceptor degeneration in mice: adeno-associated viral vector-mediated delivery of erythropoietin.

A. Auricchio, P. Colella, 2013, Methods in molecular biology.

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

N. Gomez-Ospina, G. Baldo, Vasco Meneghini, 2023, Frontiers in genome editing.

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

Monther Abu-Remaileh, N. Gomez-Ospina, J. Sarno, 2023, Research square.

CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice

N. Gomez-Ospina, J. Sarno, P. Colella, 2024, Nature communications.

Successful treatment of severe MSUD in Bckdhb−/− mice with neonatal AAV gene therapy

A. Rötig, P. de Lonlay, A. Brassier, 2023, Journal of inherited metabolic disease.

Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy.

P. Colella, 2024, Molecular diagnosis & therapy.