The past decade has seen major developments in the field of ocular gene therapy. The use of recombinant adeno-associated virus (rAAV) vectors has “jump started” the field and led to the development of very promising gene therapies for the treatment of previously untreatable genetic diseases of the eye. A number of phase I/II clinical trials have already been conducted and gene supplementation approaches for Choroideremia and RPPE 65 Leber congenital amaurosis type (LCA) 2 are now entering subsequent clinical phases. rAAV-mediated therapies for X-linked retinoschisis (XLRS), Leber hereditary optic neuropathy (LHON), the CNGA3 and CNGB3 forms of Achromatopsia and GUCY2D LCA1 have recently entered or are expected to enter the clinical phase soon, with even more clinical therapies to follow within the next few years. In addition, multiple preclinical studies highlight the potential for optogenetic strategies to treat late stage retinitis pigmentosa (RP).

Multiple important biotechnological, pharmaceutical, safety and regulatory aspects still need to be addressed as the field moves towards approval of the first ocular gene therapy drug. In particular, efforts are being made to optimize the current rAAV vector technology to increase efficiency of gene expression and specificity for the target cell type (e.g. photoreceptors or retinal pigment epithelium). Novel methods for overcoming the limitations of the packaging capacity of standard rAAV vectors must be developed in order to use this vector platform to correct inherited diseases associated with mutations in large genes. Moreover, improvements in tropism and bioavailability are also needed to allow using less invasive routes of administration. Finally, the rAAV manufacturing process needs to be optimized to meet the very high release criteria required for pharmaceutical products.

In this Research Topic we will address several of these points with Original Research Articles, Review Articles and Methods Articles covering topics ranging from basic research to translational studies. In addition to gene therapies for autosomal recessive retinal diseases we also welcome contributions on treatment options for autosomal dominant diseases and late stage degenerative diseases as well as novel biomedical applications of rAAV vectors in vision research.

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Frontiers in Neuroscience

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