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Hereditary ATTR amyloidosis

Antisense oligonucleotides and other genetic therapies made simple.
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Antisense oligonucleotides and other genetic therapies made simple.

Pract Neurol. 2018 Apr;18(2):126-131

Authors: Rossor AM, Reilly MM, Sleigh JN

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.

PMID: 29455156 [PubMed - indexed for MEDLINE]

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