About this Research Topic
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.
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.