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Vision 2017, 1(3), 22; doi:10.3390/vision1030022

Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

1
Molecular Therapy Laboratory, Murdoch University, Murdoch 6150, Australia
2
Centre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Nedlands 6009, Australia
3
Perron Institute, 4th Floor A Block, Queen Elizabeth II Medical Centre, Verdun Street, Nedlands 6009, Australia
4
Department of Ophthalmology, Royal Perth Hospital, Perth 6000, Australia
*
Author to whom correspondence should be addressed.
Received: 4 June 2017 / Revised: 24 July 2017 / Accepted: 24 August 2017 / Published: 1 September 2017
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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. View Full-Text
Keywords: alternative splicing; pre-mRNA splicing process; inherited retinal dystrophy; splicing correction; antisense oligonucleotides; retinitis pigmentosa alternative splicing; pre-mRNA splicing process; inherited retinal dystrophy; splicing correction; antisense oligonucleotides; retinitis pigmentosa
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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MDPI and ACS Style

Huang, D.; Fletcher, S.; Wilton, S.D.; Palmer, N.; McLenachan, S.; Mackey, D.A.; Chen, F.K. Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid. Vision 2017, 1, 22.

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