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Immunology

D-Index
77
Citations
31566
World Ranking
1791
National Ranking
883

Overview

Federico Mingozzi is affiliated with the Children's Hospital of Philadelphia in the United States. Their research primarily focuses on gene therapy, with specific interests in virus-based gene therapy and its applications in treating genetic and hematological disorders.

Their main fields of study include Medicine and Biochemistry, Genetics and Molecular Biology, engaging extensively with subfields such as Genetics, Molecular Biology, Oncology, Hematology, and Physiology. This interdisciplinary approach supports a broad understanding of complex biological systems relevant to gene therapy.

Mingozzi's work covers several key research topics:

  • Virus-based gene therapy research
  • CAR-T cell therapy research
  • CRISPR and Genetic Engineering
  • Hemophilia Treatment and Research
  • Lysosomal Storage Disorders Research
  • RNA Interference and Gene Delivery
  • Glycogen Storage Diseases and Myoclonus

They have contributed to numerous scientific publications with a focus on gene transfer vectors and immune responses related to gene therapy. Prominent recent papers include:

  • AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer, 2020, Molecular Therapy
  • Human Immune Responses to Adeno-Associated Virus (AAV) Vectors, 2020, Frontiers in Immunology
  • IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies, 2020, Nature Medicine
  • Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A, 2021, New England Journal of Medicine
  • Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B, 2020, Molecular Therapy

Their frequent co-authors reflect active collaboration in the field, including:

  • Giuseppe Ronzitti
  • Fanny Collaud
  • Séverine Charles
  • Klaudia Kuranda
  • Christian Leborgne

Mingozzi regularly publishes in venues that specialize in gene therapy and immunology research, such as:

  • Molecular Therapy
  • Molecular Therapy - Methods & Clinical Development
  • Blood
  • Human Gene Therapy
  • Frontiers in Immunology

Best Publications

  • Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

    Albert M. Maguire;Francesca Simonelli;Eric A. Pierce;Edward N. Pugh

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

    Manno Cs;Pierce Gf;Arruda Vr;Glader B

  • Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

    Amit C. Nathwani;Edward G.D. Tuddenham;Savita Rangarajan;Cecilia Rosales

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

    Amit C Nathwani;Ulreke M Reiss;Edward G D Tuddenham;Cecilia Rosales;Cecilia Rosales

  • Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

    Federico Mingozzi;Katherine A. High;Katherine A. High

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

    Federico Mingozzi;Katherine A. High;Katherine A. High;Katherine A. High

  • Emerging Issues in AAV-Mediated In Vivo Gene Therapy

    Pasqualina Colella;Giuseppe Ronzitti;Federico Mingozzi;Federico Mingozzi

  • CD8(+) T-cell responses to adeno-associated virus capsid in humans.

    Federico Mingozzi;Marcela V Maus;Marcela V Maus;Daniel J Hui;Denise E Sabatino

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

    Francesca Simonelli;Albert M Maguire;Albert M Maguire;Francesco Testa;Eric A Pierce

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

    Helena Costa Verdera;Klaudia Kuranda;Federico Mingozzi

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

    Federico Mingozzi;Yi-Lin Liu;Eric Dobrzynski;Antje Kaufhold

  • AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

    Jean Bennett;Jean Bennett;Manzar Ashtari;Jennifer Wellman;Kathleen A. Marshall

  • Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.

    Haiyan Jiang;Linda B. Couto;Susannah Patarroyo-White;Tongyao Liu

  • Immune responses to AAV in clinical trials.

    Federico Mingozzi;Katherine A High

  • 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.

    Amit C Nathwani;Amit C Nathwani;Cecilia Rosales;Jenny McIntosh;Ghasem Rastegarlari

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

    Federico Mingozzi;Janneke J. Meulenberg;Daniel J. Hui;Etiena Basner-Tschakarjan

  • Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

    Federico Mingozzi;Xavier M. Anguela;Xavier M. Anguela;Giulia Pavani;Yifeng Chen;Yifeng Chen

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

    E Ayuso;F Mingozzi;J Montane;X Leon

  • Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

    Jeannette Bennicelli;John Fraser Wright;John Fraser Wright;Andras Komaromy;Jonathan B Jacobs

  • Human Immune Responses to Adeno-Associated Virus (AAV) Vectors.

    Giuseppe Ronzitti;David-Alexandre Gross;Federico Mingozzi

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

    Jenny McIntosh;Jenny McIntosh;Peter J. Lenting;Cecilia Rosales;Cecilia Rosales;Doyoung Lee

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

    Federico Mingozzi;Nicole C. Hasbrouck;Etiena Basner-Tschakarjan;Shyrie A. Edmonson;Shyrie A. Edmonson

  • Assessing the potential for AAV vector genotoxicity in a murine model

    Hojun Li;Nirav Malani;Shari R. Hamilton;Alexander Schlachterman

Frequent Co-Authors

Katherine A. High
Katherine A. High Children's Hospital of Philadelphia
J. Fraser Wright
J. Fraser Wright Stanford University
Valder R. Arruda
Valder R. Arruda University of Pennsylvania
Roland W. Herzog
Roland W. Herzog Indiana University
Amit C. Nathwani
Amit C. Nathwani University College London
Anne S. De Groot
Anne S. De Groot University of Georgia
Arthur W. Nienhuis
Arthur W. Nienhuis St. Jude Children's Research Hospital
Mark A. Kay
Mark A. Kay Stanford University
Marcela V. Maus
Marcela V. Maus Harvard University
Jean Bennett
Jean Bennett University of Pennsylvania

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