(…continued from the previous post). There are several features that make the recent Adrenoleukodystrophy (ALD) gene transfer study noteworthy. 1- A New Viral Vector Debuts: this is the first successful application of HIV-derived viruses in gene transfer (lentiviruses). These vectors have various advantages over retroviruses used in other protocols. One is that, in theory, at… Continue reading More on Lenti’s, Gene Transfer and Adrenoleukodystrophy
Tag: lentiviral vectors
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… Continue reading Safe Harbor? Leukemia, Gene Transfer, and Lentiviral Vectors
Clonal Trouble
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