Clinical trials in gene therapy

Scott M. Freeman, Katharine A. Whartenby, George N. Abraham, James A. Zwiebel

Research output: Contribution to journalReview article

Abstract

Retroviral-mediated gene transfer has permitted the development of clinical protocols for the study and treatment of genetic diseases and cancer. These protocols can be divided into proposals either for gene labelling (transfer) or for gene therapy. Labelling studies include the tracking of gene-modified cells (e.g., tumor-infiltrating lymphocytes, bone marrow tumor cells) infused into the patient. Most gene therapy protocols are designed to deliver a therapeutic protein using a mature differentiated cell population (e.g., fibroblasts). A number of protocols to treat genetic diseases and cancer have been recently initiated. The approval process for the clinical studies requires a multi-agency review due to the safety and ethical concerns. While safety issues of the procedure still exist, gene therapy holds much promise as an effective treatment modality.

Original languageEnglish (US)
Pages (from-to)169-183
Number of pages15
JournalAdvanced Drug Delivery Reviews
Volume12
Issue number3
DOIs
StatePublished - Dec 1993
Externally publishedYes

Keywords

  • Gene therapy
  • Genetic disease cancer

ASJC Scopus subject areas

  • Pharmaceutical Science

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