Tag: gene transfer

  • More Gray Matter: Parkinson’s Disease and Gene Transfer

    Several groups are pursuing gene transfer strategies against Parkinson’s disease. No small task, because for these approaches to work, investigators have to deliver vectors deep inside the brain using surgery. I have previously written that early phase studies using surgical delivery press the boundaries of acceptable risk, because patients can generally manage their disease adequately-…

  • Icarus, again: Adversity in another Gene Transfer Trial

    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,…

  • Ark, Troubled Waters, and Rainbows for Gene Transfer

    This morning I awoke to a news report by National Public Radio’s Joe Palca on promising developments in gene transfer. In it, Palca provided a good account of the field’s travails, as well as some encouraging developments in the last few years. The story ended with the prediction that the coming “months and years” would…

  • Annus Mirabilis for Gene Transfer

    Time to review the year 2009 for cutting edge clinical research. For the field of gene transfer, it has been an annus mirabilis: a year that has seen very encouraging results in a wide variety of human clinical studies, as well as preclinical studies. Indeed, I regret that this blog has only been able to…

  • More on Lenti’s, Gene Transfer and Adrenoleukodystrophy

    (…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…

  • Gene Transfer and Adrenoleukodystrophy: There Will Always Be Paris

    Last week’s Science magazine reported what seems likely to count as one of gene transfer’s greatest clinical successes to date: stabilization of adrenoleukodystrophy in two boys receiving genetically modified blood stem cells. Preliminary results of this study had been presented at this summer’s American Society of Gene and Cell Therapy meeting. Adrenoleukodystrophy (ALD) is a…

  • 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…

  • Mice- Three Different Ones: Towards More Robust Preclinical Experiments

    One of the most exciting and intellectually compelling talks thus far at the American Society of Gene Therapy meeting was Pedro Lowenstein’s.  A preclinical researcher who works on gene transfer approaches to brain malignancies (among other things), Lowenstein asked the question: why do so many gene transfer interventions that look promising in the laboratory fail…

  • Who says the British Press isn’t all yellow?  “Doctors have begun trials using gene therapy to treat patients for cystic fibrosis.”  So proclaims an April 19 story in the Guardian (“Cystic fibrosis to be treated by gene therapy technology”).  “Cystic fibrosis gene cure closer,” reads a Februrary 2009 BBC headline. Details available “‘We are not…

  • 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…