Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

Jacqueline Corrigan-Curay, Odile Cohen-Haguenauer, Marina O'Reilly, Susan R. Ross, Hung Fan, Naomi Rosenberg, Nikunj Somia, Nancy King, Theodore Friedmann, Cynthia Dunbar, Alessandro Aiuti, Luigi Naldini, Christopher Baum, Christof Von Kalle, Hans Peter Kiem, Eugenio Montini, Frederic Bushman, Brian P. Sorrentino, Manuel Carrondo, Harry MalechGösta Gahrton, Robyn Shapiro, Linda Wolff, Eugene Rosenthal, Robert Jambou, John Zaia, Donald B. Kohn

Research output: Contribution to journalArticlepeer-review

34 Citations (Scopus)

Abstract

Over the past two decades, incredible progress has been made using gene therapy for inherited severe immunodeficiency disorders, such as X-linked severe combined immunodeficiency disorder (SCID-X1) and adenosine deaminase deficiency–severe combined immunodeficiency disorder (ADA-SCID).1,2,3 However, for reasons that remain unclear, gene transfer for SCID-X1 has also been associated with some cases of vector-induced leukemia whereas no cases have been seen in the ADA-SCID trials despite the common use of g-retroviral vectors. The first case was reported in a French gene transfer trial for SCID-X1. Over the next six years, an additional three cases were reported in that trial and one in a second SCID-X1 trial that enrolled a combined total of 20 subjects. Unfortunately, genotoxicity would not remain confined to SCID-X1.
Original languageEnglish
Pages (from-to)1084-1094
Number of pages11
JournalMolecular Therapy
Volume20
Issue number6
DOIs
Publication statusPublished - Jun 2012

Fingerprint Dive into the research topics of 'Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy'. Together they form a unique fingerprint.

Cite this