Two weeks ago brought good news and bad news for gene transfer. First the good news. New England Journal of Medicine beatified a new gene transfer strategy for Wiskott-Aldrich Syndrome (WAS). WAS is a primary immunodeficiency that primarily affects boys. It is thus in the same family of disorders that have been, in varying degrees,… Continue reading Icarus, again: Adversity in another Gene Transfer Trial
Tag: insertional mutagenesis
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
In Brugge / No Compassion (Part II)
Further to the therapeutic outlook on first-in-human studies at the Brugge meeting was Adrian Thrasher’s thoughtful presentation on his own X-SCID study at Great Ormand Street Hospital. Thrasher’s study was able to restore immune function in nearly all volunteers. Recently, however, his team reported a lymphoproliferative disorder like those seen in a very similar Paris… Continue reading In Brugge / No Compassion (Part II)
Just the FACS: Reprise on Insertional Mutagenesis
I‘ve just returned from the annual European Society of Gene and Cell Therapy meeting in Belgium. Lots of great material for upcoming posts. For now, I want to follow on the last posting on the leukemias in the X-SCID study. A warning: those lacking a stomach for science geek-talk might want to skip this posting.… Continue reading Just the FACS: Reprise on Insertional Mutagenesis