TY - JOUR
T1 - Overview of gene therapy clinical progress including cancer treatment with gene-modified T cells.
AU - Brenner, Malcolm K.
AU - Okur, Fatma V.
N1 - Copyright:
This record is sourced from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
PY - 2009
Y1 - 2009
N2 - It is now twenty years since the first legal gene transfer studies were approved, and there has been considerable disappointment in the slow rate of progress that followed the initial studies. Gradually, however, as the limitations of available vectors are acknowledged and overcome, and with advances in our understanding of the molecular and cell biology of genetic diseases and of cancer, unequivocal successes are now being reported. In this paper we describe the remaining major roadblocks to successful gene therapy and outline approaches to overcome them. We also illustrate how genetically modified immune system cells are already being used for the effective treatment of hematological and other malignancies, and how these approaches are being modified so that they can be effective in treating a broader range of malignancies.
AB - It is now twenty years since the first legal gene transfer studies were approved, and there has been considerable disappointment in the slow rate of progress that followed the initial studies. Gradually, however, as the limitations of available vectors are acknowledged and overcome, and with advances in our understanding of the molecular and cell biology of genetic diseases and of cancer, unequivocal successes are now being reported. In this paper we describe the remaining major roadblocks to successful gene therapy and outline approaches to overcome them. We also illustrate how genetically modified immune system cells are already being used for the effective treatment of hematological and other malignancies, and how these approaches are being modified so that they can be effective in treating a broader range of malignancies.
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U2 - 10.1182/asheducation-2009.1.675
DO - 10.1182/asheducation-2009.1.675
M3 - Review article
C2 - 20008253
AN - SCOPUS:77949475175
SN - 1520-4391
SP - 675
EP - 681
JO - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
JF - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
ER -