| خلاصه مقاله | Introduction: The current progress in ocular gene therapy is impressive after approval of first gene based therapeutic against Leber congenital amaurosis since 2017. Also, a number of clinical trials are enduring by novel targets and delivery technologies. The RNA based gene therapy approaches in eye could be safer than others because of their non-genome changing nature. By the way, RNAi is in center of RNA targeting gene therapy. In addition to Antisense and RNAi technologies, CRISPER mediated RNA editing has emerged and enabled scientific community to initiate developing targeted RNA therapeutics and precise single nucleotide modifications more efficient and specified. The focus of this review is on recent advances of RNA-targeting biosystems for ocular disorders and will consider the implications of RNA level therapeutics in order to generate future treatments of ever-increasing eye disease.
Material and methods: A comprehensive searching and reviewing the biomedical literature was performed since 2000, using gene therapy, RNAi, ocular disease, eye, Crisper Cas 9 & 13 keywords. The non-relevant articles were excluded and tried to depict an overview of advancements in ocular gene therapy at RNA level.
Results and discussion: Present review demonstrates a variety of RNA targeting biotherapeutics developed to be candidates for treating ocular disease. MiRNA mimic and inhibitors and siRNA therapeutics have been studied in many ocular disease-based research projects and clinical trials showing hopeful and encouraged outcomes. So, they are expected to add on our knowledge and come to clinical practice. The exciting novel aspect of ocular RNA therapeutics is gene editing CRISPR-Cas and its derivatives. In this regard, ocular gene therapy against acquired or inherited disease at post transcriptional level could take the advantages of CRISPR-Cas13 platform by RNA bases editing, single or multiple RNA knockdown, and inducible interference. Given the prevalence of Age-related macular degeneration (AMD), Diabetic retinopathy (DR), and other blinding eye conditions, generation of RNA targeting therapeutics due to their more safe and efficient potential is extremely recommended at this time. |