The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute

Terence R. Flotte, Eric Daniels, Janet Benson, Jeneé M. Bevett-Rose, Kenneth Cornetta, Margaret Diggins, Julie Johnston, Susan Sepelak, Johannes C.M. Van Der Loo, James M. Wilson, Cheryl L. McDonald

Research output: Contribution to journalReview article

1 Scopus citations

Abstract

Over a 10-year period, the Gene Therapy Resource Program (GTRP) of the National Heart Lung and Blood Institute has provided a set of core services to investigators to facilitate the clinical translation of gene therapy. These services have included a preclinical (research-grade) vector production core; current Good Manufacturing Practice clinical-grade vector cores for recombinant adeno-associated virus and lentivirus vectors; a pharmacology and toxicology core; and a coordinating center to manage program logistics and to provide regulatory and financial support to early-phase clinical trials. In addition, the GTRP has utilized a Steering Committee and a Scientific Review Board to guide overall progress and effectiveness and to evaluate individual proposals. These resources have been deployed to assist 82 investigators with 172 approved service proposals. These efforts have assisted in clinical trial implementation across a wide range of genetic, cardiac, pulmonary, and blood diseases. Program outcomes and potential future directions of the program are discussed.

Original languageEnglish (US)
Pages (from-to)178-186
Number of pages9
JournalHuman Gene Therapy Clinical Development
Volume28
Issue number4
DOIs
StatePublished - Dec 1 2017

Keywords

  • adeno-associated virus
  • clinical trial
  • Gene therapy
  • lentivirus
  • preclinical
  • vector

ASJC Scopus subject areas

  • Genetics(clinical)

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    Flotte, T. R., Daniels, E., Benson, J., Bevett-Rose, J. M., Cornetta, K., Diggins, M., Johnston, J., Sepelak, S., Van Der Loo, J. C. M., Wilson, J. M., & McDonald, C. L. (2017). The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute. Human Gene Therapy Clinical Development, 28(4), 178-186. https://doi.org/10.1089/humc.2017.170