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Inherited retinal disease therapies targeting precursor messenger ribonucleic acid

Huang, D., Fletcher, S., Wilton, S., Palmer, N., McLenachan, S., Mackey, D. and Chen, F. (2017) Inherited retinal disease therapies targeting precursor messenger ribonucleic acid. Vision, 1 (3). p. 22.

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Abstract

Inherited retinal diseases are an extremely diverse group of genetically and phenotypically heterogeneous conditions characterized by variable maturation of retinal development, impairment of photoreceptor cell function and gradual loss of photoreceptor cells and vision. Significant progress has been made over the last two decades in identifying the many genes implicated in inherited retinal diseases and developing novel therapies to address the underlying genetic defects. Approximately one-quarter of exonic mutations related to human inherited diseases are likely to induce aberrant splicing products, providing opportunities for the development of novel therapeutics that target splicing processes. The feasibility of antisense oligomer mediated splice intervention to treat inherited diseases has been demonstrated in vitro, in vivo and in clinical trials. In this review, we will discuss therapeutic approaches to treat inherited retinal disease, including strategies to correct splicing and modify exon selection at the level of pre-mRNA. The challenges of clinical translation of this class of emerging therapeutics will also be discussed.

Item Type: Journal Article
Murdoch Affiliation: Centre for Comparative Genomics
Publisher: MDPI
Copyright: © 2017 by the authors. Licensee MDPI, Basel, Switzerland
URI: http://researchrepository.murdoch.edu.au/id/eprint/42210
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