Bottlenecks in development of retinal therapeutic post-transcriptional gene silencing agents.
Development of post-transcriptional gene silencing (PTGS) agents for therapeutic purposes is an immense challenge in modern biology. Established technologies used to knockdown a specific target RNA and its cognate protein: antisense, ribozyme, RNAi, all conditionally depend upon an initial, critical annealing event of the PTGS ligand to a target RNA. In this review we address the nature of the bottlenecks, emphasizing the biocomplexity of target RNA structure, that currently limit PTGS therapeutic development. We briefly review existing and emerging technologies designed to release these constraints to realize the potential of PTGS agents in gene based therapies.
Sullivan
Jack M
JM
Department of Ophthalmology, SUNY, University at Buffalo, Veterans Administration Western New York Healthcare System, Medical Research, Building 20, Room 245, 3495 Bailey Avenue, Buffalo, NY 14215, USA. jackmsullivanmdphd@yahoo.com
Yau
Edwin H
EH
Taggart
R Thomas
RT
Butler
Mark C
MC
Kolniak
Tiffany A
TA
D018390:Gene Targeting / Q000379:methods; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D034622:RNA Interference; D012323:RNA Processing, Post-Transcriptional / Q000235:genetics; D012333:RNA, Messenger / Q000235:genetics; D034741:RNA, Small Interfering / Q000235:genetics; D012164:Retinal Diseases / Q000628:therapy*; D017299:Rod Opsins / Q000235:genetics
D012333:RNA, Messenger; D034741:RNA, Small Interfering; D017299:Rod Opsins