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  • Robin J. Ziegler, Scott D. Bercury, +8 authors Seng H. Cheng
  • Biology, Medicine
  • Human gene therapy
  • 2008 (First Published: 1 June 2008)
  • The availability of a murine model of Pompe disease has enabled an evaluation of the relative merits of various therapeutic paradigms, including gene therapy. We report here that administration of aContinue Reading
  • Jennifer Rha, Stephanie K Jones, +17 authors Anita H. Corbett
  • Biology, Medicine
  • Human molecular genetics
  • 2017 (First Published: 1 October 2017)
  • A number of mutations in genes that encode ubiquitously expressed RNA-binding proteins cause tissue specific disease. Many of these diseases are neurological in nature revealing critical roles forContinue Reading