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Biology and Biochemistry

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90
Citations
27048
World Ranking
2450
National Ranking
1299

Medicine

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91
Citations
27473
World Ranking
11842
National Ranking
6072

Overview

Terence R. Flotte is affiliated with the University of Massachusetts Chan Medical School in the United States. Their research primarily focuses on areas within Biochemistry, Genetics, and Molecular Biology, with significant contributions to Medicine as well.

The main subfields of study for this scientist include Molecular Biology, Genetics, Physiology, Oncology, and Cardiology and Cardiovascular Medicine. Their scientific work covers a broad range of topics, with emphasis on:

  • Virus-based gene therapy research
  • CRISPR and Genetic Engineering
  • RNA Interference and Gene Delivery
  • CAR-T cell therapy research
  • Neurogenetic and Muscular Disorders Research
  • Biomedical Ethics and Regulation
  • Viral Infections and Immunology Research

Terence R. Flotte has contributed to various prestigious journals. Their most frequent publication venues include:

  • Human Gene Therapy
  • Molecular Therapy
  • bioRxiv (Cold Spring Harbor Laboratory)
  • New England Journal of Medicine
  • Molecular Therapy - Methods & Clinical Development

Recent papers by Flotte reflect important topics in gene therapy and related biomedical fields. Notable publications include:

  • Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice, 2021, Nature Communications
  • Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy, 2023, New England Journal of Medicine
  • SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS, 2020, New England Journal of Medicine
  • Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy, 2020, Human Gene Therapy
  • Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer, 2023, BioDrugs

The scientist collaborates frequently with several coauthors, indicating active research partnerships. Prominent frequent collaborators include:

  • Guangping Gao
  • Allison M. Keeler
  • Meghan Blackwood
  • Alisha M. Gruntman
  • Mohammed Salman Shazeeb

Terence R. Flotte's body of work spans fundamental and applied aspects of gene therapy, genetic engineering, and molecular medicine, encompassing both experimental research and considerations on biomedical ethics and regulation.

Best Publications

  • Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

    William W. Hauswirth;Tomas S. Aleman;Shalesh Kaushal;Artur V. Cideciyan

  • Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

    Artur V. Cideciyan;Tomas S. Aleman;Sanford L. Boye;Sharon B. Schwartz

  • Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

    Terence R. Flotte;Sandra A. Afione;Carol Conrad;Sharon A. McGrath

  • Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AAV2 Vectors with Altered Tropism

    Pei Wu;Wu Xiao;Thomas Conlon;Jeffrey Hughes

  • Adeno-associated virus vectors for gene therapy

    T R Flotte;B J Carter

  • VIRUS ADENO-ASSOCIADO COM SEQUENCIAS DA REPETICAO TERMINAL INVERTIDA COMO PROMOTOR PARA A TRANSFERENCIA IN VIVO DE UM GENE DE CFTR FUNCIONAL

    Carter Barrie J;Flotte Terrence;Afione Sandra;Solow Rikki

  • Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR

    Marie Egan;Terence Flotte;Terence Flotte;Sandra Afione;Rikki Solow

  • Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter

    Terence R. Flotte;Sandra A. Afione;Rikki Solow;Mitchell L. Drumm

  • Clinical gene therapy using recombinant adeno-associated virus vectors

    Christian Mueller;Terence R. Flotte

  • Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

    Mark L. Brantly;Jeffrey D. Chulay;Lili Wang;Christian Mueller

  • Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.

    Terence R. Flotte;Bruce C. Trapnell;Margaret Humphries;Brenna Carey

  • Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

    Sihong Song;Michael Morgan;Tamir Ellis;Amy Poirier

  • A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease

    Terence Flotte;Barrie Carter;Carol Conrad;William Guggino

  • Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors

    A Donsante;C Vogler;N Muzyczka;JM Crawford

  • In vivo model of adeno-associated virus vector persistence and rescue

    Sandra A. Afione;Carol K. Conrad;William G. Kearns;Suryaprabha Chunduru

  • Phase I Trial of Intranasal and Endobronchial Administration of a Recombinant Adeno-Associated Virus Serotype 2 (rAAV2)-CFTR Vector in Adult Cystic Fibrosis Patients: A Two-Part Clinical Study

    Terence R. Flotte;Pamela L. Zeitlin;Thomas C. Reynolds;Alison E. Heald

  • Gene Expression from Adeno-associated Virus Vectors in Airway Epithelial Cells

    Terence R. Flotte;Rikki Solow;Roland A. Owens;Sandra Afione

  • α-1 Antitrypsin Inhibits Caspase-3 Activity, Preventing Lung Endothelial Cell Apoptosis

    Irina Petrache;Iwona Fijalkowska;Terry R. Medler;Jarrett Skirball

  • Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration.

    Terence R. Flotte;Sandra A. Afione;Pamela L. Zeitlin

  • A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

    John A. Wagner;Ilynn B. Nepomuceno;Anna H. Messner;Mary Lynn Moran

Frequent Co-Authors

Christian Mueller
Christian Mueller University of Massachusetts Chan Medical School
Barry J. Byrne
Barry J. Byrne University of Florida
Mark A. Atkinson
Mark A. Atkinson University of Florida
William B. Guggino
William B. Guggino Johns Hopkins University
Martha Campbell-Thompson
Martha Campbell-Thompson University of Florida
Guangping Gao
Guangping Gao University of Massachusetts Chan Medical School
Barrie J. Carter
Barrie J. Carter National Institutes of Health
William W. Hauswirth
William W. Hauswirth University of Florida
James M. Wilson
James M. Wilson University of Pennsylvania
Pedro E. Cruz
Pedro E. Cruz University of Florida

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