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Antisense oligonucleotides and other genetic therapies made simple

Rossor, A; Reilly, M; Sleigh, JN; (2018) Antisense oligonucleotides and other genetic therapies made simple. Practical Neurology , 18 (2) pp. 126-131. 10.1136/practneurol-2017-001764. Green open access

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Abstract

Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors. This review provides a brief overview of the main types of genetic therapies, with a focus on antisense oligonucleotides (ASOs) and RNA interference (RNAi). We use examples of new genetic therapies for spinal muscular atrophy, Duchenne muscular dystrophy and familial amyloid polyneuropathy to highlight the different mechanisms of action of ASOs and RNAi.

Type: Article
Title: Antisense oligonucleotides and other genetic therapies made simple
Open access status: An open access version is available from UCL Discovery
DOI: 10.1136/practneurol-2017-001764
Publisher version: https://doi.org/10.1136/practneurol-2017-001764
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases
URI: https://discovery.ucl.ac.uk/id/eprint/10043999
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