Gene therapy applications to transfusion medicine

Eric A. Gehrie, Alexey Bersenev, Emanuela Bruscia, Diane Krause, Wade L. Schulz

Research output: Chapter in Book/Report/Conference proceedingChapter


This chapter focuses on the possible applications of gene therapy to transfusion medicine. Gene therapy falls within the purview of transfusion medicine when it requires the infusion of either gene-modified cells or direct administration of vectors that contain the therapeutic genetic material. The chapter summarizes the salient features of the viral vectors. It discusses nonviral techniques that are under development for gene therapy. Replication-competent viruses develop either by recombination of the constituent parts of the vector system with endogenous viral-sequences in the vector packaging cell lines or by activation of endogenous proviral-sequences. The chapter also discusses three types of genetic diseases such as hemophilia B, hemophilia A and hemoglobinopathies, where hemophilia B and hemophilia A are caused by a deficiency in factor IX and factor VIII respectively. It further describes gene-editing approaches such as transcription activator-like effector nucleases, zinc finger nucleases, and clustered regularly interspaced short palindromic repeats.

Original languageEnglish (US)
Title of host publicationRossi’s Principles of Transfusion Medicine
Number of pages4
ISBN (Electronic)9781119013020
ISBN (Print)9781119012993
StatePublished - Apr 15 2016
Externally publishedYes


  • Clustered regularly interspaced short palindromic repeats
  • Gene therapy
  • Hemoglobinopathies
  • Hemophilia a
  • Hemophilia b
  • Retroviral vectors
  • Transcription activator-like effector nucleases
  • Transfusion medicine
  • Zinc finger nucleases

ASJC Scopus subject areas

  • Medicine(all)


Dive into the research topics of 'Gene therapy applications to transfusion medicine'. Together they form a unique fingerprint.

Cite this