Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

Di Huang; Sue Fletcher; Steve D Wilton; Norman Palmer; Samuel McLenachan; David A Mackey; Fred K Chen

doi:10.3390/vision1030022

Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

Di Huang, Sue Fletcher, Steve D Wilton, Norman Palmer, Samuel McLenachan, David A Mackey, Fred K Chen

Research output: Contribution to journal › Review article › peer-review

7 Citations (Scopus)

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.

Original language	English
Article number	22
Journal	Vision
Volume	1
Issue number	3
DOIs	https://doi.org/10.3390/vision1030022
Publication status	Published - 1 Sept 2017

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.3390/vision1030022Licence: CC BY

Cite this

@article{5b33d90c582f4078bffa61526d19ab9e,

title = "Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid",

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.",

author = "Di Huang and Sue Fletcher and Wilton, {Steve D} and Norman Palmer and Samuel McLenachan and Mackey, {David A} and Chen, {Fred K}",

year = "2017",

month = sep,

day = "1",

doi = "10.3390/vision1030022",

language = "English",

volume = "1",

journal = "Vision",

issn = "2411-5150",

publisher = "MDPI AG",

number = "3",

}

TY - JOUR

T1 - Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

AU - Huang, Di

AU - Fletcher, Sue

AU - Wilton, Steve D

AU - Palmer, Norman

AU - McLenachan, Samuel

AU - Mackey, David A

AU - Chen, Fred K

PY - 2017/9/1

Y1 - 2017/9/1

N2 - 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.

AB - 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.

U2 - 10.3390/vision1030022

DO - 10.3390/vision1030022

M3 - Review article

C2 - 31740647

SN - 2411-5150

VL - 1

JO - Vision

JF - Vision

IS - 3

M1 - 22

ER -

Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

Abstract

UN SDGs

Access to Document

Fingerprint

Cite this