Jude Samulski PhD work in gene therapy improves outcomes for DMD

Duchenne Muscular Dystrophy was one of the early targets for gene therapy, and as early as 1993, using a viral vector with an inserted dystrophin gene was shown to work in mice models.  But since then, progress has been slow, partly in finding the right delivery method, and to some extent, failures of other gene therapy trials.  This piece from the University of North Carolina, Chapel Hill, is about a success against DMD.  The vector was an adeno-associated virus that carried the dystrophin gene—and the fact that the gene is 500 times larger than the vector illustrates why this problem was difficult to overcome.   MORE

Image Credit: UNC News