Gene Therapy

Studies from P.A. Radcliffe and colleagues provide new data on gene therapy

Published in Gene Therapy Weekly, May 8th, 2008

"Effective gene therapy for haemophilia A necessitates a vector system that is not subject to a pre-existing immune response, has adequate coding capacity, gives long-term expression and preferably can target non-dividing cells. Vector systems based on lentiviruses such as equine infectious anaemia virus (EIAV) fulfil these criteria for the delivery of factor VIII (FVIII)," scientists in Oxford, the United Kingdom report.

"We have found that B domain-deleted (BDD) FVIII protein inhibits functional viral particle production when co-expressed with the EIAV vector system. Although particle numbers (as measured by reverse transcriptase activity) are near normal, RNA genome...

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Source: Gene Therapy Weekly (2008-05-08)

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