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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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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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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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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.1245-1262

2019

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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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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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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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Overcoming preexisting humoral immunity to AAV using capsid decoys

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

2013

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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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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

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

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

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

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

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

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

Overcoming preexisting humoral immunity to AAV using capsid decoys

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)

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