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Frederic D. Bushman
J Clin Invest. 2007;
117(8):2083
doi:10.1172/JCI32949
Abstract |
Full text
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L
ong-term correction of genetic diseases requires permanent integration of therapeutic genes into chromosomes of affected cells. Retroviral vectors are the most widely used delivery vehicles because of their efficiency and precision of integration. However, retroviral integration can take place at a variety of chromosomal sites, and examples have been reported of integration of therapeutic vectors activating oncogenes and causing cancer in patients. This issue of the JCI presents three articles that update successful human gene therapy trials and furthermore evaluate the sites of integration in cells from treated patients, including samples from individuals experiencing serious adverse events following therapy (see the related articles beginning on pages 2225, 2233, and 2241).
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(36)
| Title and authors |
Publication |
Year |
Methods in Enzymology
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|
Gene Transfer Vectors for Clinical Application
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2012 |
Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell
Luca Biasco, Alessandro Ambrosi, Danilo Pellin, Cynthia Bartholomae, Immacolata Brigida, Maria Grazia Roncarolo, Clelia Di Serio, Christof von Kalle, Manfred Schmidt, Alessandro Aiuti
|
EMBO Mol Med
|
2011 |
Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning
Marshall W Huston, Niek P van Til, Trudi P Visser, Shazia Arshad, Martijn H Brugman, Claudia Cattoglio, Ali Nowrouzi, Yuedan Li, Axel Schambach, Manfred Schmidt, Christopher Baum, Christof von Kalle, Fulvio Mavilio, Fang Zhang, Mike P Blundell, Adrian J Thrasher, Monique MA Verstegen, Gerard Wagemaker
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Mol Ther
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2011 |
Harper's Textbook of Pediatric Dermatology
Julie V. Schaffer, Amy S. Paller
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Harper's Textbook of Pediatric Dermatology
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2011 |
State-of-the-art gene-based therapies: the road ahead
Mark A. Kay
|
Nat Rev Genet
|
2011 |
Risk factors in the development of stem cell therapy.
Carla A Herberts, Marcel S G Kwa, Harm P H Hermsen
|
J Transl Med
|
2011 |
Gene therapy for lysosomal storage disorders
Angela Gritti
|
Expert Opin. Biol. Ther.
|
2011 |
Oligo/Polynucleotide-Based Gene Modification: Strategies and Therapeutic Potential
R. Geoffrey Sargent, Soya Kim, Dieter C. Gruenert
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Oligonucleotides
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2011 |
Recombinant Adeno-Associated Virus-Mediated Gene Transfer for the Potential Therapy of Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
Jared N. Silver, Melissa Elder, Thomas Conlon, Pedro Cruz, Amy J. Wright, Arun Srivastava, Terence R. Flotte
|
Human Gene Therapy
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2011 |
Treatment of advanced leukemia in mice with mRNA engineered T cells
David Maxwell Barrett, Yangbing Zhao, Xiaojun Liu, Shuguang Jiang, Carmine Carpenito, Michael Kalos, Richard G Carroll, Carl H June, Stephan A Grupp
|
Human Gene Therapy
|
2011 |
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