siRNA Therapeutics in Ocular Diseases

The field of RNAi therapeutics has quickly adapted to the treatment of ocular diseases. Although the eye provides a unique system for the delivery of siRNAs, its complex structure and composition fostered the development of novel strategies for efficient gene silencing in the target compartment. Mor...

Full description

Saved in:
Bibliographic Details
Published inMethods in molecular biology (Clifton, N.J.) Vol. 2282; p. 417
Main Authors Moreno-Montañés, Javier, Bleau, Anne-Marie, Martínez, Tamara, Vargas, Beatriz, González, María Victoria, Jiménez, Ana Isabel
Format Journal Article
LanguageEnglish
Published United States 01.01.2021
Subjects
Online AccessGet more information

Cover

Loading…
More Information
Summary:The field of RNAi therapeutics has quickly adapted to the treatment of ocular diseases. Although the eye provides a unique system for the delivery of siRNAs, its complex structure and composition fostered the development of novel strategies for efficient gene silencing in the target compartment. Moreover, anterior and posterior segments differ in their multiple drug barriers and clearance mechanisms. This chapter summarizes the recent achievements in terms of routes of administration, chemical modifications, and delivery systems for siRNAs that specifically apply to eye disorders. Methods employed for siRNA detection/quantitation in ocular tissues are also described, together with safety concerns that need to be addressed to fulfill regulatory requirements of new drug approval. Even though RNAi therapies for ocular diseases have not yet translated into patient care, we document herein the rising number of candidate drugs currently under preclinical or clinical development.
ISSN:1940-6029
DOI:10.1007/978-1-0716-1298-9_23