Ocular Gene Therapy: A Literature Review With Focus on Current Clinical Trials
Open Access
- 24 September 2022
- journal article
- review article
- Published by Springer Science and Business Media LLC in Cureus
- Vol. 14 (9), e29533
- https://doi.org/10.7759/cureus.29533
Abstract
Gene therapy has been one of the most researched topics in the last decade. It has now become a revolutionized therapeutic tool of modern medicine. Gene therapy is the alteration of the defective gene involved in the disease process in the host cells. It delivers therapeutic genetic information via modified viral or non-viral vectors. Ocular gene therapy, in particular, has progressed in treating inherited retinal diseases since the eye is a favourable organ for gene therapy development. The advantage of the eye as a target for gene therapy is attributed to its easy accessibility and blood-ocular barrier. Several ongoing clinical trials are investigating various gene therapies for other ocular diseases, including neovascular age-related macular degeneration, retinitis pigmentosa (RP), Usher syndrome, glaucoma, and several others. However, there are challenges such as ocular inflammation and humoral response, infection by the viral vectors, and insertional mutagenesis. These limitations depend on several factors; whether viral or non-viral vectors are used, which viral vectors were used, the route of administration, whether subretinal, intravitreal, or suprachoroidal, and the dose of vectors and the target tissue. These complications may lead to therapeutic failure and vision loss due to intraocular inflammation. This review aims to summarize existing knowledge about ocular gene therapy and the associated limitations we face, with a special focus on a few ongoing clinical trials.This publication has 30 references indexed in Scilit:
- The CRISPR/Cas9 system for gene editing and its potential application in pain research2016
- Non Viral Vectors in Gene Therapy- An OverviewJournal of Clinical and Diagnostic Research, 2015
- Gene Editing ofCCR5in Autologous CD4 T Cells of Persons Infected with HIVThe New England Journal of Medicine, 2014
- Small‐interfering RNAs (siRNAs) as a promising tool for ocular therapyBritish Journal of Pharmacology, 2013
- Corneal Gene Therapy: Basic Science and Translational PerspectiveThe Ocular Surface, 2013
- Ocular gene delivery using lentiviral vectorsGene Therapy, 2011
- Gene Transfer for Neovascular Age-Related Macular DegenerationHuman Gene Therapy, 2011
- Adenoviral Vector-Delivered Pigment Epithelium-Derived Factor for Neovascular Age-Related Macular Degeneration: Results of a Phase I Clinical TrialHuman Gene Therapy, 2006
- Immune response following intraocular delivery of recombinant viral vectorsGene Therapy, 2003
- Adenovirus–mediated retinoblastoma gene therapy suppresses spontaneous pituitary melanotroph tumors in Rb+/− miceNature Medicine, 1996