Part 4-Gene Therapy: A Researcher’s Perspective
RARECast - Ein Podcast von RARECast - Donnerstags
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P.J. Brooks, program director in the Office of Rare Disease Research at the National Center for Advancing Translational Sciences, discusses the PaVe-GT program, the potential for developing a toolkit of plug-and-play vectors, and how this can alter the cost of developing gene therapies for ultra-rare and individual patients.