World's Best Scientists 2026 revealed!

D-Index & Metrics

Molecular Biology

D-Index
70
Citations
28162
World Ranking
1397
National Ranking
709

Overview

Federico Mingozzi is affiliated with the University of Pennsylvania in the United States. Their research spans primarily the fields of Medicine and Biochemistry, Genetics and Molecular Biology, with a focus on Genetics, Molecular Biology, Oncology, Hematology, and Physiology.

The scientist's work concentrates notably on 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, and glycogen storage diseases and myoclonus.

Frequent publication venues for Federico Mingozzi include:

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

Some of the recent papers authored or co-authored by Mingozzi are:

  • "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

Frequent collaborators in their research include:

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

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

  • 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

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

    Albert M. Maguire;Albert M. Maguire;Katherine A. High;Katherine A. High;Alberto Auricchio;J. Fraser Wright;J. Fraser Wright

  • 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

  • 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

    Jean Bennett;Jean Bennett;Jennifer Wellman;Kathleen A Marshall;Sarah McCague

  • 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

  • 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

  • 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

  • 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

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

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

Frequent Co-Authors

Olivier Benveniste
Olivier Benveniste Sorbonne University
Olivier Boyer
Olivier Boyer University of Rouen
Hildegard Büning
Hildegard Büning Hannover Medical School
Francesco Bernardi
Francesco Bernardi University of Ferrara
Henry J. Kaplan
Henry J. Kaplan University of Louisville
Constance L. Cepko
Constance L. Cepko Harvard University
Mark A. Kay
Mark A. Kay Stanford University
Alan H. Beggs
Alan H. Beggs Harvard Medical School
George M. Church
George M. Church Harvard University
Michio Hirano
Michio Hirano Columbia University

If you think any of the details on this page are incorrect, let us know.

Report an issue

We appreciate your kind effort to assist us to improve this page, it would be helpful providing us with as much detail as possible in the text box below:

Related Online Degrees & Career Pathways

The field of molecular biology connects to a wide range of dynamic career paths and educational opportunities. Many students consider online degrees for greater flexibility, allowing them to balance studies with work or life commitments. For those interested in related STEM fields, a bsc mathematics online can complement molecular biology by strengthening analytical and quantitative skills valuable in research and data analysis.

If you are drawn to creativity and communication, a graphic design online degree offers tools to help visualize scientific ideas or build impactful presentations. Some may be interested in bridging molecular science with architecture; exploring online architecture programs could open opportunities in biotech facilities design and lab planning.

For professionals seeking a sector change, such as educators wanting to transition to healthcare, resources on career change for teachers provide helpful guidance. Ultimately, these diverse online degree options and career pathways can empower you to personalize your journey in molecular biology and beyond.

Best Scientists Citing Federico Mingozzi

Trending Scientists