Emerging Concepts for RNA Therapeutics for Inherited Retinal Disease

Spencer M Moore; Dorota Skowronska-Krawczyk; Daniel L Chao

doi:10.1007/978-3-030-27378-1_14

Emerging Concepts for RNA Therapeutics for Inherited Retinal Disease

Adv Exp Med Biol. 2019:1185:85-89. doi: 10.1007/978-3-030-27378-1_14.

Authors

Spencer M Moore¹, Dorota Skowronska-Krawczyk², Daniel L Chao³

Affiliations

¹ Medical Scientist Training Program, School of Medicine, University of California, San Diego, La Jolla, CA, USA.
² Department of Ophthalmology, Shiley Eye Institute, University of California, San Diego, La Jolla, CA, USA.
³ Department of Ophthalmology, Shiley Eye Institute, University of California, San Diego, La Jolla, CA, USA. dlchao@ucsd.edu.

PMID: 31884593
DOI: 10.1007/978-3-030-27378-1_14

Abstract

Inherited retinal diseases (IRD) encompass a wide spectrum of hereditary blindness with significant genetic heterogeneity. Therapeutics regulating gene expression on an RNA level have significant promise for treating IRD. In this review, we review the molecular basis of oligonucleotide therapeutics such as ribozymes, RNA interference (RNAi), antisense oligonucleotides (ASO), CRISPRi/a, and their applications to treatments of IRD.

Keywords: Antisense oligonucleotide; CRISPRi/a; Inherited retinal disease; RNA interference; Ribozyme.

Publication types

Review

MeSH terms

Clustered Regularly Interspaced Short Palindromic Repeats
Humans
Oligonucleotides, Antisense / therapeutic use
RNA / therapeutic use*
RNA Interference
RNA, Catalytic / therapeutic use
Retinal Diseases / therapy*

Substances

Oligonucleotides, Antisense
RNA, Catalytic
RNA

Grants and funding

R01 EY027011/EY/NEI NIH HHS/United States