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Federico Mingozzi

French Institute of Health and Medical Research
Université Paris-Saclay

48

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1162

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Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome.

2021 Molecular Therapy
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A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia.

2021 Science Advances
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Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses.

2021 Science Translational Medicine
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IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies.

2020 Nature Medicine
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A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

2020 Molecular therapy. Methods & clinical development
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Early Phase Clinical Immunogenicity of Valoctocogene roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

2020 Molecular Therapy
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AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

2020 Molecular Therapy
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In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery.

2020 Scientific Reports
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Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

2020 Scientific Reports
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Rescue of advanced Pompe disease in mice with hepatic expression of secretable acid α-glucosidase

2020 Molecular Therapy
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