Focus10TH ANNIVERSARY SERIES

Wrangling RNA: Antisense oligonucleotides for neurological disorders

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Science Translational Medicine  25 Sep 2019:
Vol. 11, Issue 511, eaay2069
DOI: 10.1126/scitranslmed.aay2069

Figures

  • Fig. 1 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.

    CREDIT: A. KITTERMAN/SCIENCE TRANSLATIONAL MEDICINE

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