Probiotic therapies involve the use of living microorganisms to treat or prevent disease. A number of creative applications are being developed for treatment of colitis, prevention of dental caries, and control of infectious disease. Some of these applications are likely to involve genetically modified strains of bacteria. Look for more of these in the next… Continue reading Probiotics take a knock in the gut?
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In Utero: Gene Transfer Nirvana?
The February 2008 issue of Molecular Therapy has an editorial by Charles Coutelle defending in utero gene transfer (that is, the application of gene transfer to fetuses). Coutelle makes several attractive arguments in its favor. Among them: that many parents opt against abortion after receiving a prenatal diagnosis, and that in utero gene transfer would… Continue reading In Utero: Gene Transfer Nirvana?
Why the Title “Lost in Translation?”
The title of this blog derives from that of my book on the ethics of human gene transfer research, which is contracted with Cambridge University Press. As the title suggests, the translation of gene transfer into clinical application has not gone as smoothly as predicted. The word “lost” is not intended to suggest incompetence, or… Continue reading Why the Title “Lost in Translation?”
The Scope of this Blog
Though my primary focus is on gene transfer, I will occasionally wander into other areas of translational pharmaceutical research, including cell transfer (also sometimes called “cell therapy”), monoclonal antibodies, small-molecule drugs that target novel biological mechanisms, or large-molecule drugs. These types of translational clinical research raise a similar cluster of issues– namely, high degrees of… Continue reading The Scope of this Blog
Why not call it Gene Therapy?
What I term “gene transfer” is often also called “gene therapy.” I prefer the former, which is based less on aspiration than fact. That is, therapy implies validation for efficacy. There are, at best, only two examples of gene transfer interventions where efficacy seems well established. Use of the term “gene therapy” potentially misleads members… Continue reading Why not call it Gene Therapy?
What is Gene Transfer?
In this blog, I define gene transfer as the use of genetic materials or genetically modified organisms for therapeutic or research purposes. This is a much broader definition than typical. For instance, the American Society of Gene Therapy defines gene therapy as “…an approach to treating disease by either modifying the expressions of an individual’s… Continue reading What is Gene Transfer?
Introduction to Lost in Translation
Within the past six months, a fifth patient developed a lympho-proliferative disorder in an X-SCID gene transfer trial. The RAC has all but cleared AAV vector as a causal agent in the death of Jolee Mohr in a rheumatoid arthritis study. A Merck HIV-vaccine trial involving adenoviral vectors was terminated after a data safety monitoring… Continue reading Introduction to Lost in Translation