Literature DB >> 12835668

Occurrence of leukaemia following gene therapy of X-linked SCID.

Donald B Kohn1, Michel Sadelain, Joseph C Glorioso.   

Abstract

Recombinant viral vectors have allowed gene transfer to be developed as a promising approach to the treatment of genetic diseases. Recently, gene therapy of children with X-linked severe combined immune deficiency resulted in impressive levels of immune reconstitution--a triumph that was later overshadowed by the development of leukaemia in two patients. What were the causes of this cancer, and how can the therapeutic benefits of gene therapy be achieved while minimizing risk to the patient?

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Year:  2003        PMID: 12835668     DOI: 10.1038/nrc1122

Source DB:  PubMed          Journal:  Nat Rev Cancer        ISSN: 1474-175X            Impact factor:   60.716


  88 in total

1.  Identification of synthetic endothelial cell-specific promoters by use of a high-throughput screen.

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2.  Hype and public trust in science.

Authors:  Zubin Master; David B Resnik
Journal:  Sci Eng Ethics       Date:  2011-11-02       Impact factor: 3.525

Review 3.  Safe harbours for the integration of new DNA in the human genome.

Authors:  Michel Sadelain; Eirini P Papapetrou; Frederic D Bushman
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Review 4.  Hematopoietic stem cell engineering at a crossroads.

Authors:  Isabelle Rivière; Cynthia E Dunbar; Michel Sadelain
Journal:  Blood       Date:  2011-11-17       Impact factor: 22.113

Review 5.  Gene therapy for ocular diseases.

Authors:  Melissa M Liu; Jingsheng Tuo; Chi-Chao Chan
Journal:  Br J Ophthalmol       Date:  2010-08-23       Impact factor: 4.638

Review 6.  Cellular endocytosis and gene delivery.

Authors:  Jennifer E Ziello; Yan Huang; Ion S Jovin
Journal:  Mol Med       Date:  2010-02-03       Impact factor: 6.354

7.  Specific insertions of zinc finger domains into Gag-Pol yield engineered retroviral vectors with selective integration properties.

Authors:  Kwang-il Lim; Ryan Klimczak; Julie H Yu; David V Schaffer
Journal:  Proc Natl Acad Sci U S A       Date:  2010-06-28       Impact factor: 11.205

8.  Foe turned friend: multiple functional roles attributable to hyper-activating stem cell factor receptor mutant in regeneration of the haematopoietic cell compartment.

Authors:  S Pati; O P Kalra; A Mukhopadhyay
Journal:  Cell Prolif       Date:  2011-02       Impact factor: 6.831

Review 9.  Cardiovascular gene therapy for myocardial infarction.

Authors:  Maria C Scimia; Anna M Gumpert; Walter J Koch
Journal:  Expert Opin Biol Ther       Date:  2013-12-16       Impact factor: 4.388

10.  A capsid-modified helper-dependent adenovirus vector containing the beta-globin locus control region displays a nonrandom integration pattern and allows stable, erythroid-specific gene expression.

Authors:  Hongjie Wang; Dmitry M Shayakhmetov; Tobias Leege; Michael Harkey; Qiliang Li; Thalia Papayannopoulou; George Stamatoyannopolous; André Lieber
Journal:  J Virol       Date:  2005-09       Impact factor: 5.103

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