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Kapil Bharti; Induced to cure: Using iPS cells to develop autologous cell therapy for degenerative eye diseases. Journal of Vision 2014;14(15):1. doi: https://doi.org/10.1167/14.15.1.
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The recent success with embryonic stem (ES) cell derived retinal pigment epithelium (RPE) has provided hope for a treatment for degenerative eye diseases. Induced pluripotent stem (iPS) cells are an alternate and an autologous source of stem cells potentially with fewer immune-challenges as compared to ES cells. Using a developmentally guided differentiation protocol we have developed fully polarized RPE tissue from iPS cells. The RPE monolayer along with its secreted ECM and the scaffold form a tissue that well mimics the native tissue. This tissue has been functionally authenticated in vitro for its ability to perform several key RPE functions and is being tested in animal models. We have begun manufacturing for a Phase I Investigational New Drug (IND) to transplant autologous iPS cell derived RPE in patients in advanced Geographic Atrophy stage of age-related macular degeneration (AMD), one of the leading blinding diseases in the US. We propose to develop National Institutes of Health Clinical Center as an “alpha-stem cell clinic” with the capability to recruit patients, manufacture clinical-grade autologous iPS cell derived RPE tissue, perform transplantation, do patient care, and be able to transfer technology to academic and private sectors. We suggest that our open access model with complete access to the entire IND package will reduce redundant efforts in the field, foster public-private partnerships, and help move ocular regenerative medicine field forward.
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