Tag: SCID

  • Safe Harbor? Leukemia, Gene Transfer, and Lentiviral Vectors

    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…

  • 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…

  • Departing Milano Stazione? ADA-SCID and Gene Transfer

    Greetings after a hiatus for teaching, grants, committees, book deadlines, wiping runny noses, and more. Much has happened since my last posting, and in the next two or three weeks, I hope to catch up. First item on the agenda is a Jan 29 report in New England Journal of Medicine (NEJM) describing successful reconstitution of…

  • 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…

  • 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.…

  • 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…