Gene Therapy

Studies conducted at A. Aiuti et al on gene therapy recently published

Published in Gene Therapy Weekly, August 6th, 2009

"Gene therapy is a highly attractive strategy for many types of inherited disorders of the immune system. Adenosine deaminase (ADA) deficient-severe combined immunodeficiency (SCID) has been the target of several clinical trials based on the use of hematopoietic stem/progenitor cells engineered with retroviral vectors," researchers in Milan, Italy report.

"The introduction of a low intensity conditioning regimen has been a crucial factor in achieving stable engrafment of hematopoietic stem cells and therapeutic levels of ADA-expressing cells. Recent studies have demonstrated that gene therapy for ADA-SCID has favorable safety profile and is effective in restoring normal...

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Source: Gene Therapy Weekly (2009-08-06)

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