Biomaterial Database

Emerging Concepts for RNA Therapeutics for Inherited Retinal Disease. 2019

Spencer M Moore, and Dorota Skowronska-Krawczyk, and Daniel L Chao

Medical Scientist Training Program, School of Medicine, University of California, San Diego, La Jolla, CA, USA.

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.

UI	MeSH Term	Description	Entries
D012164	Retinal Diseases	Diseases involving the RETINA.	Disease, Retinal,Diseases, Retinal,Retinal Disease
D006801	Humans	Members of the species Homo sapiens.	Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D012313	RNA	A polynucleotide consisting essentially of chains with a repeating backbone of phosphate and ribose units to which nitrogenous bases are attached. RNA is unique among biological macromolecules in that it can encode genetic information, serve as an abundant structural component of cells, and also possesses catalytic activity. (Rieger et al., Glossary of Genetics: Classical and Molecular, 5th ed)	RNA, Non-Polyadenylated,Ribonucleic Acid,Gene Products, RNA,Non-Polyadenylated RNA,Acid, Ribonucleic,Non Polyadenylated RNA,RNA Gene Products,RNA, Non Polyadenylated
D016337	RNA, Catalytic	RNA that has catalytic activity. The catalytic RNA sequence folds to form a complex surface that can function as an enzyme in reactions with itself and other molecules. It may function even in the absence of protein. There are numerous examples of RNA species that are acted upon by catalytic RNA, however the scope of this enzyme class is not limited to a particular type of substrate.	Catalytic RNA,Ribozyme,Ribozymes
D016376	Oligonucleotides, Antisense	Short fragments of DNA or RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.	Anti-Sense Oligonucleotide,Antisense Oligonucleotide,Antisense Oligonucleotides,Anti-Sense Oligonucleotides,Anti Sense Oligonucleotide,Anti Sense Oligonucleotides,Oligonucleotide, Anti-Sense,Oligonucleotide, Antisense,Oligonucleotides, Anti-Sense
D034622	RNA Interference	A gene silencing phenomenon whereby specific dsRNAs (RNA, DOUBLE-STRANDED) trigger the degradation of homologous mRNA (RNA, MESSENGER). The specific dsRNAs are processed into SMALL INTERFERING RNA (siRNA) which serves as a guide for cleavage of the homologous mRNA in the RNA-INDUCED SILENCING COMPLEX. DNA METHYLATION may also be triggered during this process.	Gene Silencing, Post-Transcriptional,Post-Transcriptional Gene Silencing,Co-Suppression,Cosuppression,Posttranscriptional Gene Silencing,RNA Silencing,RNAi,Co Suppression,Gene Silencing, Post Transcriptional,Gene Silencing, Posttranscriptional,Gene Silencings, Posttranscriptional,Interference, RNA,Post Transcriptional Gene Silencing,Post-Transcriptional Gene Silencings,Silencing, Post-Transcriptional Gene
D064112	Clustered Regularly Interspaced Short Palindromic Repeats	Repetitive nucleic acid sequences that are principal components of the archaeal and bacterial CRISPR-CAS SYSTEMS, which function as adaptive antiviral defense systems.	CRISPR Arrays,CRISPR Clusters,CRISPR Elements,CRISPR Loci,CRISPR Locus,CRISPR Sequences,CRISPR Spacer Sequences,CRISPR Spacers,CRISPR-Cas Loci,CRISPRs,Clustered Regularly Interspaced Short Palindromic Repeat,Array, CRISPR,Arrays, CRISPR,CRISPR,CRISPR Array,CRISPR Cas Loci,CRISPR Cluster,CRISPR Element,CRISPR Sequence,CRISPR Spacer,CRISPR Spacer Sequence,CRISPR-Cas Locus,Cluster, CRISPR,Clusters, CRISPR,Element, CRISPR,Elements, CRISPR,Loci, CRISPR,Loci, CRISPR-Cas,Locus, CRISPR,Locus, CRISPR-Cas,Sequence, CRISPR,Sequence, CRISPR Spacer,Sequences, CRISPR,Sequences, CRISPR Spacer,Spacer Sequence, CRISPR,Spacer Sequences, CRISPR,Spacer, CRISPR,Spacers, CRISPR