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Previous Volume 350:913-922 February 26, 2004 Number 9 Next

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The emerging field of gene therapy promises to yield exciting new treatments for a range of illnesses. Most forms of gene therapy use viral delivery mechanisms, most commonly retroviruses,¹ and one condition in which this approach has corrected a genetic defect is X-linked severe combined immunodeficiency (SCID).² X-linked SCID is characterized by the lack of specific types of lymphocytes (Figure 1A and Figure 1B)^7,12 owing to the absence of the common subunit of the interleukin-2 receptor (IL2Rc). This subunit of the interleukin-2 cytokine receptor is a signaling molecule that is a component of receptors for several other . . . [Full Text of this Article]

A Major Adverse Effect of Viral Gene Therapy

LMO2, a Quintessential Chromosomal Translocation Oncogene

LMO2 as a Target of Retroviral Activation

Role of IL2Rc in Leukemogenesis

Chromosomal Translocation or Retroviral Activation plus Other Events

Conclusions

Source Information

From the Rotary Bone Marrow Research Laboratory, Royal Melbourne Hospital, Melbourne, Victoria, Australia (M.P.M.); and the Medical Research Council Laboratory of Molecular Biology, Cambridge, United Kingdom (T.H.R.).

Related Letters:

LMO2 and Gene Therapy for Severe Combined Immunodeficiency
Fischer A., Abina S. H.-B., Thrasher A., von Kalle C., Cavazzana-Calvo M., Stone B. D., Rabbitts T. H., McCormack M. P.
Extract | Full Text | PDF  
N Engl J Med 2004; 350:2526-2527, Jun 10, 2004. Correspondence

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