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

James E. Summerton

doi:10.1007/978-1-4939-6817-6_1

Morpholino Oligomers pp 1-15

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

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

Authors
Authors and affiliations

James E. SummertonEmail author

Protocol

First Online:: 01 April 2017

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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.

Key words

Morpholino oligomers Antisense Anti-gene History Delivery

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Authors and Affiliations

James E. Summerton
- 1
Email author

1.Gene Tools, LLCPhilomathUSA

About this protocol

Cite this protocol as:: 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

DOI 10.1007/978-1-4939-6817-6_1
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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Invention and Early History of Morpholinos: From Pipe Dream to Practical Products

Abstract

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