List of works - Federico Mingozzi

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

scientific article published on 20 July 2016

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

scientific article

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

scientific article

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

scientific article published on 08 June 2009

AAV2 gene therapy readministration in three adults with congenital blindness.

scientific article

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

scientific article published on 10 December 2011

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

scientific article

Assessing the potential for AAV vector genotoxicity in a murine model

scientific article

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

scientific article published on 11 May 2009

Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions

scientific article

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

scientific article published on 16 January 2013

Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade

scientific article

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

scientific article published on 23 January 2012

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

scientific article

Host and vector-dependent effects on the risk of germline transmission of AAV vectors

scientific article

IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression

scientific article published on 02 July 2013

Identification of mouse AAV capsid-specific CD8+ T cell epitopes

scientific article published on 2 November 2005

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

scientific article published on 01 June 2020

Immune responses to AAV in clinical trials

scientific article published on October 2007

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

scientific article published on 17 April 2013

Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector

scientific article

In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity

scientific article published on 02 September 2012

Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products

scientific article published on 15 September 2016

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer ⬇️

scientific article published on May 1, 2003

Innate and Adaptive Immune Response in Fabry Disease

scientific article published on 18 February 2015

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

scientific article

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

scientific article

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

scientific article

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

scientific article published on 16 July 2013

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

scientific article

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

scientific article

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

scientific article

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis

scientific article published on 24 June 2015

Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

scholarly article

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

scientific article published in November 2017

Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer

scientific article

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

scientific article published on 30 June 2016

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

scientific article

Safety and efficacy of gene transfer for Leber's congenital amaurosis

scientific article

Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

scientific article

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

scientific article

Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors

scientific article published on 11 March 2020

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

scientific article (publication date: March 2006)

Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease

scientific article published on October 2012

Th1 response and systemic treg deficiency in inclusion body myositis.

scientific article

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

scientific article published on 20 February 2013

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

scientific article

Tregitope peptides: the active pharmaceutical ingredient of IVIG?

scientific article published on 25 December 2013

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

scientific article

List of works by Federico Mingozzi

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

AAV2 gene therapy readministration in three adults with congenital blindness.

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Assessing the potential for AAV vector genotoxicity in a murine model

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

Host and vector-dependent effects on the risk of germline transmission of AAV vectors

IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression

Identification of mouse AAV capsid-specific CD8+ T cell epitopes

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Immune responses to AAV in clinical trials

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector

In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity

Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer ⬇️

Innate and Adaptive Immune Response in Fabry Disease

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

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis

Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

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

Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease

Th1 response and systemic treg deficiency in inclusion body myositis.

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

Tregitope peptides: the active pharmaceutical ingredient of IVIG?

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy