Gene therapy enters the pharma market: The short story of a long journey

Abstract

Approximately 20 years ago, gene therapy was first introduced when Michael Blaese and colleagues applied ex vivo modified autologous T cells to children suffering from adenosine deaminase deficiency (ADA-SCID), a Mendelian genetic error that causes severe combined immunodeficiency syndrome (Blaese et al, 1995). The investigators used retroviral vectors; the… (More)
DOI: 10.1002/emmm.201202291

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