A few further observations from the American Society of Gene Therapy Meeting… A recurrent theme in this blog is the frequency with which novel research fields encounter safety problems that confound laboratory predictions. One presentation at the 2009 ASGT meeting brought this point home. Recall my entry on May 12 discussing various refinements to retroviral… Continue reading Safe Harbor? Leukemia, Gene Transfer, and Lentiviral Vectors
Tag: retroviral vectors
Yellow Light on Gene Transfer Studies
Among the greatest heartbreaks in the field of gene transfer have been problems encountered in trials involving a rare, hereditary immune disorder, X-SCID (known popularly as “Bubble Boy” syndrome). As is well known, a team of researchers based in Paris– and then in London– successfully reversed severe immunodeficiencies in 20 or so children using retroviral… Continue reading Yellow Light on Gene Transfer Studies
Burst Bubbles
Among the greatest traumas for gene transfer was the development of leukemias in several children participating in trials using retroviral vectors against X-linked Severe Combined Immune Deficiency (X-SCID– also known as “bubble boy syndrome”). About 20 or so children have had their immune systems fully restored by this gene transfer strategy. Tragically, however, five children… Continue reading Burst Bubbles