Fig. 1 Advancing ASO therapy for neurological disorders.

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Presentation transcript:

Fig. 1 Advancing ASO therapy for neurological disorders. Advancing ASO therapy for neurological disorders. Future advances in ASO therapy for neuromuscular and neurodegenerative disorders will depend on both technical advances (left) and improved screening and biomarker strategies for identifying patients during the earliest stages of disease (right). Development of next-generation ASO compounds will arise from advances in oligonucleotide chemistry, including stereoselective synthesis, coupled with technologies that solve the challenges of intracellular ASO delivery and delivery across the blood-brain barrier. Last, the ability to assess the true clinical effectiveness of ASO therapies will be driven not only by presymptomatic genetic screening of neonates or at-risk family members but also by development of more robust clinical trial outcome measures, including development of digital technologies and devices that will have implications for reductions in clinical trial size, duration, and ultimately cost. Kevin Talbot and Matthew J. A. Wood Sci Transl Med 2019;11:eaay2069 Copyright © 2019 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works