In a first application of gene therapy for the treatment of Wiskott-Aldrich Syndrome, blood forming bone marrow cells have been corrected by gene transfer in Hannover, Germany.
A team led by Professor Christoph Klein has succeeded in correcting symptoms of this rare, inherited immunodeficiency in 9 out of 10 children in a clinical trial. One patient did not receive a sufficient number of cells, and one patient unfortunately developed an acute T cell leukemia related to the treatment. In an article published in the New England Journal of Medicine this week, Klein and colleagues report on details of their trial’s two first patients with four years of follow-up. “We are delighted about the possibility to offer a new form of therapy to this group of patients” says Klein. “However, we have to proceed very carefully, because the inherent risks of the retrovirus vector technology can produce serious side effects, as occurred in one of our patients.” Earlier this year, Professor Klein received the renowned Leibniz Award of the German Research Foundation (DFG) for his work in pediatric immunology.The Wiskott-Aldrich Syndrome
Prior to clinical application of this gene therapy Klein’s interdisciplinary team has closely worked with philosophers and bioethicists. “For our WAS children without suitable transplant donors, gene therapy offers a chance to return to a normal life, as Felix’s example shows,” explains Klein.Care for Rare Foundation
Further information: Professor Christoph Klein, email@example.com, phone +49 511 532-6711
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