About this Research Topic
For many ocular diseases, gene expression control by gene augmentation or gene inhibition strategies are revolutionizing the field of ocular drug delivery by proposing compounds with long term effects. Viral vectors allowed the first proof-of-concept to efficiently restore visual functions in patients affected by an inherited retinal dystrophy, such as LCA2. Since this fantastic progress several clinical trials are ongoing, but few data are available to precisely describe the treatment efficacy and the advances made in the methodology to reliably produce clean and efficient vectors with the optimal excipient to deliver it. Multiple vector designs are emerging and a clear methodology description is necessary to allow the community of the gene therapy field to exchange knowledge on the use or ameliorations of the most promising tools. A similar statement can be made for technologies using non-viral vector delivery or antisense-oligonucleotides (ASO).
The present Research Topic aims to present articles on the state-of-the-art of the drug delivery controlling gene expression from a point of view of deep methodology description and treatment efficacy. Dual vectors, gene editing, capsid optimization, non-viral gene delivery, and ASO strategies, among others, will be presented and discussed in the context of the important progresses made as well as the expected future developments. The goal is to propose a panel of technologies in ocular gene therapy to foster researchers to use the appropriate tool, among various applications in the field, in order to potentially treat many ocular diseases which have no cure.
Each ocular disease has its specificity, necessitating an appropriate approach to prevent tissue degradation and restore normal cell homeostasis. In consequence, different tools are necessary to induce adequate actions. Vectors to deliver large genes or to obtain local and transient gene expression were successful in large animal studies or in first clinical studies, whereas intravitreal gene delivery via viral vectors is still in development to avoid potential deleterious effects of subretinal injections. ASOs have shown surprising long-term effects after a single injection opening new perspectives for certain targets. Gene editing is exploiting a variety of techniques to transfer the CRISPR/Cas9 system which is starting to produce promising results in animal models, suggesting that future improvements may soon be satisfactory to implement such technology in clinical trials. The current issue should disclose the existing and future strategies in drug delivery to correct gene expression for ocular diseases, allowing researchers to choose the optimal application for each disease.
The present Research Topic aims to present articles on the state-of-the-art of the drug delivery controlling gene expression from a point of view of deep methodology description and treatment efficacy. Dual vectors, gene editing, capsid optimization, non-viral gene delivery, and ASO strategies, among others, will be presented and discussed in the context of the important progresses made as well as the expected future developments. The goal is to propose a panel of technologies in ocular gene therapy to foster researchers to use the appropriate tool, among various applications in the field, in order to potentially treat many ocular diseases which have no cure.
Each ocular disease has its specificity, necessitating an appropriate approach to prevent tissue degradation and restore normal cell homeostasis. In consequence, different tools are necessary to induce adequate actions. Vectors to deliver large genes or to obtain local and transient gene expression were successful in large animal studies or in first clinical studies, whereas intravitreal gene delivery via viral vectors is still in development to avoid potential deleterious effects of subretinal injections. ASOs have shown surprising long-term effects after a single injection opening new perspectives for certain targets. Gene editing is exploiting a variety of techniques to transfer the CRISPR/Cas9 system which is starting to produce promising results in animal models, suggesting that future improvements may soon be satisfactory to implement such technology in clinical trials. The current issue should disclose the existing and future strategies in drug delivery to correct gene expression for ocular diseases, allowing researchers to choose the optimal application for each disease.
Keywords: Ocular Disease, Gene Therapy, CRISPR, Viral Vectors, Retinal, Non-Viral Vectors
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