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Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration.
Stem Cells. 2009 Sep; 27(9):2126-35.SC

Abstract

Assessments of safety and efficacy are crucial before human ESC (hESC) therapies can move into the clinic. Two important early potential hESC applications are the use of retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration and Stargardt disease, an untreatable form of macular dystrophy that leads to early-onset blindness. Here we show long-term functional rescue using hESC-derived RPE in both the RCS rat and Elov14 mouse, which are animal models of retinal degeneration and Stargardt, respectively. Good Manufacturing Practice-compliant hESC-RPE survived subretinal transplantation in RCS rats for prolonged periods (>220 days). The cells sustained visual function and photoreceptor integrity in a dose-dependent fashion without teratoma formation or untoward pathological reactions. Near-normal functional measurements were recorded at >60 days survival in RCS rats. To further address safety concerns, a Good Laboratory Practice-compliant study was carried out in the NIH III immune-deficient mouse model. Long-term data (spanning the life of the animals) showed no gross or microscopic evidence of teratoma/tumor formation after subretinal hESC-RPE transplantation. These results suggest that hESCs could serve as a potentially safe and inexhaustible source of RPE for the efficacious treatment of a range of retinal degenerative diseases.

Authors+Show Affiliations

Casey Eye Institute, Oregon Health and Science University, Portland, Oregon 97239, USA.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

19521979

Citation

Lu, Bin, et al. "Long-term Safety and Function of RPE From Human Embryonic Stem Cells in Preclinical Models of Macular Degeneration." Stem Cells (Dayton, Ohio), vol. 27, no. 9, 2009, pp. 2126-35.
Lu B, Malcuit C, Wang S, et al. Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration. Stem Cells. 2009;27(9):2126-35.
Lu, B., Malcuit, C., Wang, S., Girman, S., Francis, P., Lemieux, L., Lanza, R., & Lund, R. (2009). Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration. Stem Cells (Dayton, Ohio), 27(9), 2126-35. https://doi.org/10.1002/stem.149
Lu B, et al. Long-term Safety and Function of RPE From Human Embryonic Stem Cells in Preclinical Models of Macular Degeneration. Stem Cells. 2009;27(9):2126-35. PubMed PMID: 19521979.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration. AU - Lu,Bin, AU - Malcuit,Christopher, AU - Wang,Shaomei, AU - Girman,Sergej, AU - Francis,Peter, AU - Lemieux,Linda, AU - Lanza,Robert, AU - Lund,Raymond, PY - 2009/6/13/entrez PY - 2009/6/13/pubmed PY - 2009/12/16/medline SP - 2126 EP - 35 JF - Stem cells (Dayton, Ohio) JO - Stem Cells VL - 27 IS - 9 N2 - Assessments of safety and efficacy are crucial before human ESC (hESC) therapies can move into the clinic. Two important early potential hESC applications are the use of retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration and Stargardt disease, an untreatable form of macular dystrophy that leads to early-onset blindness. Here we show long-term functional rescue using hESC-derived RPE in both the RCS rat and Elov14 mouse, which are animal models of retinal degeneration and Stargardt, respectively. Good Manufacturing Practice-compliant hESC-RPE survived subretinal transplantation in RCS rats for prolonged periods (>220 days). The cells sustained visual function and photoreceptor integrity in a dose-dependent fashion without teratoma formation or untoward pathological reactions. Near-normal functional measurements were recorded at >60 days survival in RCS rats. To further address safety concerns, a Good Laboratory Practice-compliant study was carried out in the NIH III immune-deficient mouse model. Long-term data (spanning the life of the animals) showed no gross or microscopic evidence of teratoma/tumor formation after subretinal hESC-RPE transplantation. These results suggest that hESCs could serve as a potentially safe and inexhaustible source of RPE for the efficacious treatment of a range of retinal degenerative diseases. SN - 1549-4918 UR - https://www.unboundmedicine.com/medline/citation/19521979/Long_term_safety_and_function_of_RPE_from_human_embryonic_stem_cells_in_preclinical_models_of_macular_degeneration_ L2 - https://doi.org/10.1002/stem.149 DB - PRIME DP - Unbound Medicine ER -