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High Throughput Screening in Duchenne Muscular Dystrophy: From Drug Discovery to Functional Genomics

Department of Neurology, David Geffen School of Medicine, University of California Los Angeles, 710 Westwood Plaza, Los Angeles, CA 90095, USA
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Biology 2014, 3(4), 752-780; https://doi.org/10.3390/biology3040752
Received: 8 September 2014 / Revised: 28 October 2014 / Accepted: 30 October 2014 / Published: 14 November 2014
(This article belongs to the Special Issue Screening for Biologically Active Compounds)
Centers for the screening of biologically active compounds and genomic libraries are becoming common in the academic setting and have enabled researchers devoted to developing strategies for the treatment of diseases or interested in studying a biological phenomenon to have unprecedented access to libraries that, until few years ago, were accessible only by pharmaceutical companies. As a result, new drugs and genetic targets have now been identified for the treatment of Duchenne muscular dystrophy (DMD), the most prominent of the neuromuscular disorders affecting children. Although the work is still at an early stage, the results obtained to date are encouraging and demonstrate the importance that these centers may have in advancing therapeutic strategies for DMD as well as other diseases. This review will provide a summary of the status and progress made toward the development of a cure for this disorder and implementing high-throughput screening (HTS) technologies as the main source of discovery. As more academic institutions are gaining access to HTS as a valuable discovery tool, the identification of new biologically active molecules is likely to grow larger. In addition, the presence in the academic setting of experts in different aspects of the disease will offer the opportunity to develop novel assays capable of identifying new targets to be pursued as potential therapeutic options. These assays will represent an excellent source to be used by pharmaceutical companies for the screening of larger libraries providing the opportunity to establish strong collaborations between the private and academic sectors and maximizing the chances of bringing into the clinic new drugs for the treatment of DMD. View Full-Text
Keywords: dystrophin; utrophin; DMD; mdx; AONs; stop codon read-through; siRNA; RTC; Galgt2; HTS dystrophin; utrophin; DMD; mdx; AONs; stop codon read-through; siRNA; RTC; Galgt2; HTS
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Gintjee, T.J.; Magh, A.S.; Bertoni, C. High Throughput Screening in Duchenne Muscular Dystrophy: From Drug Discovery to Functional Genomics. Biology 2014, 3, 752-780.

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