Cellular Therapy for Degenerative Retinopathies

Description

This template was adapted from the original submission. Edits were made to enhance scientific accuracy, optimal usability and/or to meet industry-leading design standards for science communication. Degenerative retinopathies show progressive loss of retinal cells. To stop this process, a patient’s skin sample is cultured and fibroblasts are reprogrammed with Yamanaka Factors into induced Pluripotent Stem Cells (iPSC). Genetic analysis allows for guide RNAs generation that will target the mutated gene for correction using CRISPR-Cas9. Treated iPSC are differentiated to retinal pigmented epithelia (RPE) or photoreceptors and injected into the subretinal space, where they will settle.

Acknowledgements

References

Riera M. et.al. (2019) Generation of an induced pluripotent stem cell line (FRIMOi002-A) from a retinitis pigmentosa patient carrying compound heterozygous mutations in USH2A gene. . Stem Cell Res.. https://www.sciencedirect.com/science/article/pii/S1873506119300145?via%3Dihub Ben M’Barek K. et.al. (2019) Developing Cell-Based Therapies for RPE-Associated Degenerative Eye Diseases. Adv Exp Med Biol.. https://link.springer.com/chapter/10.1007%2F978-3-030-28471-8_3 Gonzalez-Cordero A. et.al. (2017) Recapitulation of Human Retinal Development from Human Pluripotent Stem Cells Generates Transplantable Populations of Cone Photoreceptors. Stem Cell Reports. https://www.cell.com/stem-cell-reports/fulltext/S2213-6711(17)30332-6?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2213671117303326%3Fshowall%3Dtrue Maeda A et.al. (2019) Gene and Induced Pluripotent Stem Cell Therapy for Retinal Diseases. Annu Rev Genomics Hum Genet.. https://www.annualreviews.org/doi/10.1146/annurev-genom-083118-015043

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