In the February 20th edition of the New York Times, Reed Abelson reports a “New Tack on a Muscle Disease.” The “new tack” is, in fact, a scaling back of ambition in muscular dystrophy (MD) research. Since identification of various genetic mutations that cause muscular dystrophies, gene transfer has been a logical goal. But because… Continue reading Still Life with Duchenne Muscular Dystrophy
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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?