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Morpholino Oligomers pp 1–15Cite as

Invention and Early History of Morpholinos: From Pipe Dream to Practical Products

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 1565))

Abstract

Beginning with my concept in 1969 to treat disease at the nucleic acid level using antisense nucleic acids, antisense has evolved to the current Morpholino oligos. Morpholinos have been the dominant gene knockdown system in developmental biology. Lack of delivery technologies has limited their use in adult animals (including humans), though alteration in muscles in Duchenne muscular dystrophy (DMD) allows delivery into adult muscle. Morpholinos are currently in Phase 3 clinical trials for DMD and a Morpholino oligo for skipping dystrophin exon 51 has been approved by the US FDA. With improved delivery techniques, such as those in development at Gene Tools, therapeutic Morpholinos for many difficult-to-treat diseases will be possible. Initial applications are expected to be custom cocktails of delivery-enabled Morpholinos for treating cancers.

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Author information

Authors and Affiliations

  1. Gene Tools, LLC, 1001 Summerton Way, Philomath, OR, 97370, USA

    James E. Summerton

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  1. James E. Summerton
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Correspondence to James E. Summerton .

Editor information

Editors and Affiliations

  1. Department of Biomedical Sciences College of Veterinary Medicine, Oregon State University, Corvallis, Oregon, USA

    Hong M. Moulton

  2. Gene Tools, LLC, Philomath, Oregon, USA

    Jon D. Moulton

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Summerton, J.E. (2017). Invention and Early History of Morpholinos: From Pipe Dream to Practical Products. In: Moulton, H., Moulton, J. (eds) Morpholino Oligomers. Methods in Molecular Biology, vol 1565. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-6817-6_1

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  • DOI: https://doi.org/10.1007/978-1-4939-6817-6_1

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-6815-2

  • Online ISBN: 978-1-4939-6817-6

  • eBook Packages: Springer Protocols

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