The retina is a vital component of the visual system, responsible for converting light into neural signals. Retinal diseases such as age-related macular degeneration, retinitis pigmentosa, and diabetic retinopathy can lead to irreversible vision loss, presenting significant challenges for affected individuals and healthcare systems. Despite advances in medical and surgical interventions, the restoration of retinal function remains elusive. However, the field of regenerative medicine, including cell and gene therapy, offers promising avenues for retinal regeneration. Gene therapy can augment the absence of a functional gene or even be used to employ gene editing techniques to prevent or reverse vision loss. Retinal organoids that are derived from pluripotent stem cells provide a source of differentiated cell populations that can replace damaged retinal cells and thereby restore retinal architecture and function. In some cases, stem cells and gene therapy approaches provide platforms for drug discovery and identification of new drug targets to treat retinal degenerative diseases.

This Research Topic focuses on the latest advances in retinal regeneration through stem cell therapy and related innovations. By collecting different article types, this collection aims to foster an in-depth understanding of current challenges, novel methodologies, and future directions in the field of retinal regeneration.

Areas to be covered might include, but are not limited to:

o Stem Cell Sources and Differentiation: Exploration of various stem cell types, including embryonic stem cells, induced pluripotent stem cells, and adult stem cells, in retinal regeneration. Comparative studies on the differentiation capabilities and efficiency of different stem cell types into retinal cell lineages and retinal organoids.

o Gene Therapy: Novel vector designs, non-viral vectors, ex-vivo gene therapy, large animal models, human trials. This would include only cutting-edge technologies and not articles using existing vectors to treat mouse models of human disease.

o Innovative Delivery Systems: Development of bioengineering and nanotechnology-based approaches for optimizing stem cell and gene therapy delivery to the retina. Examination of scaffolds, hydrogels, and biomimetic materials designed to enhance cell survival, integration, and function.

o Retinal Microenvironment and Integration: Studies focusing on the retinal microenvironment's role in stem cell survival and integration. Insights into the adaptation and integration of stem cells within the existing retinal tissue.

o Applications of Gene Editing and Advanced Techniques: Utilization of gene editing tools, such as CRISPR/Cas9, to enhance stem cell functionality and safety. Innovative techniques to improve the precision and effectiveness of stem cell therapies in retinal regeneration.

o Use of gene transfer and stem cells in retinal drug discovery: Use of stem cells, organoids, and gene transfer to perform drug screening in vitro and in novel animal models. Cell-based pre-clinical assays, in-process and release assays, and safety studies.

Topic Editor Dr. Alan Marmorstein is the founder and former managing member of LAgen Laboratories LLC and founder of Seeing Medicines Inc (SMI), both closed in 2023. The other Topic Editors declare no competing interests with regard to the Research Topic subject.

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Advancing Retinal Regeneration: The Role of Stem Cells in Therapy and Innovation