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Photo-activated raster scanning thermal imaging at sub-diffraction resolution

Nature communications, 2019-12, Vol.10 (1), p.5523-9, Article 5523

2019

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Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

Gene therapy, 2013-04, Vol.20 (4), p.417-424

2013

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High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

Gene therapy, 2010-04, Vol.17 (4), p.503-510

2010

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Exosome-associated AAV vector as a robust and convenient neuroscience tool

Gene therapy, 2016-04, Vol.23 (4), p.380-392

2016

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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

Journal of inherited metabolic disease, 2024-01, Vol.47 (1), p.119-134

2024

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μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure

Scientific reports, 2017-12, Vol.7 (1), p.17468-13, Article 17468

2017

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AAV Immunogenicity: A Matter of Sensitivity

Molecular therapy, 2018-10, Vol.26 (10), p.2335-2336

Mingozzi, Federico

2018

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I.11AAV vectors and the host immune system: a complicated relationship

Neuromuscular disorders : NMD, 2019-10, Vol.29, p.S121-S122

Mingozzi, F.

2019

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AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

Molecular therapy, 2020-03, Vol.28 (3), p.723-746

2020

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Peripheral immunization of mice to produce antibodies against NMDA receptor as a potential approach to create a rodent model of schizophrenia

European neuropsychopharmacology, 2016-10, Vol.26, p.S271-S272

2016

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Author Correction: μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure

Scientific reports, 2018-04, Vol.8 (1), p.6314-1, Article 6314

2018

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Exosome-associated AAV vector as a robust and convenient neuroscience tool

Gene therapy, 2016-11, Vol.23 (11), p.819-819

2016

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Gene Therapy for Pompe Disease: The Time is now

Human gene therapy, 2019-10, Vol.30 (10), p.1245

2019

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Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

Annual review of virology, 2017-09, Vol.4 (1), p.511-534

2017

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Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

Gene therapy, 2005-10, Vol.12 (19), p.1453-1464

2005

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P1213 : Adeno-associated virus vector-mediated liver gene therapy for Crigler-Najjar syndrome

Journal of hepatology, 2015-04, Vol.62, p.S812-S812

2015

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Erratum: Exosome-associated AAV vector as a robust and convenient neuroscience tool

Gene therapy, 2016-11, Vol.23 (11), p.819-819

2016

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T.P.21

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.870-871

2014

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Cytokine and chemokine profiling shows rapid activation of the immune system following enzyme replacement therapy in Pompe disease

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S187-S187

2015

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Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy

Neuromuscular disorders : NMD, 2016-10, Vol.26, p.S209-S209

2016

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Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

The New England journal of medicine, 2014-11, Vol.371 (21), p.1994-2004

2014

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Natural history and functional status of patients with myotubular myopathy enrolled in a prospective and longitudinal study

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S274-S274

2015

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Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study

Neuromuscular disorders : NMD, 2015-10, Vol.25, p.S274-S274

2015

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Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)

Neuromuscular disorders : NMD, 2015, Vol.25, p.S315-S315

2015

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Overcoming preexisting humoral immunity to AAV using capsid decoys

Science translational medicine, 2013-07, Vol.5 (194), p.194ra92

2013

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Photo-activated raster scanning thermal imaging at sub-diffraction resolution

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

Exosome-associated AAV vector as a robust and convenient neuroscience tool

Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure

AAV Immunogenicity: A Matter of Sensitivity

I.11AAV vectors and the host immune system: a complicated relationship

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

Peripheral immunization of mice to produce antibodies against NMDA receptor as a potential approach to create a rodent model of schizophrenia

Author Correction: μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure

Exosome-associated AAV vector as a robust and convenient neuroscience tool

Gene Therapy for Pompe Disease: The Time is now

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

P1213 : Adeno-associated virus vector-mediated liver gene therapy for Crigler-Najjar syndrome

Erratum: Exosome-associated AAV vector as a robust and convenient neuroscience tool

T.P.21

Cytokine and chemokine profiling shows rapid activation of the immune system following enzyme replacement therapy in Pompe disease

Prolonged benefit from systemic rAAV8 in a canine model of myotubular myopathy

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Natural history and functional status of patients with myotubular myopathy enrolled in a prospective and longitudinal study

Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study

Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)

Overcoming preexisting humoral immunity to AAV using capsid decoys

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