Using human induced pluripotent stem cells to treat retinal disease

Borooah, Shyamanga; Phillips, M. Joseph; Bilican, Bilada; Wright, Alan F.; Wilmut, Ian; Chandran, Siddharthan; Gamm, David M.; Dhillon, Baljean

doi:10.1016/j.preteyeres.2013.09.002

Cited by 61 publications

(45 citation statements)

References 165 publications

(196 reference statements)

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“…Whereas RPE replacement alone may be used for specific disease indications, transplantation of photoreceptors-as retinal sheet or as suspension of dissociated cells-is required after extensive photoreceptor degeneration. Retinal neurons, including photoreceptors, have been generated from human iPSCs by various laboratories worldwide (for reviews, see Borooah et al 112 and Chen et al 113 ), but so far cell transplantation to restore neural retina is restricted to animal models. Prior groundbreaking studies in mice revealed that the ontogenetic stage of transplanted cells is crucial for successful integration into the adult host retina and recovery of vision.…”

Section: Cell Transplantation Using Human Pscsmentioning

confidence: 99%

Let There Be Light: Gene and Cell Therapy for Blindness

et al. 2016

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Section: Cell Transplantation Using Human Pscsmentioning

confidence: 99%

Let There Be Light: Gene and Cell Therapy for Blindness

et al. 2016

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“…The hESC RPE in animals improved functional performance, after comparing with controls, and significant similarity compared to what is found in adult humans [6,8,9] . The United States FDA recently approved a Ⅰ/Ⅱ clinical trial, currently ongoing with hESC, for the treatment of GA and Stargardt's macular dystrophy [7,19] . Pan et al [12] , reported the first subretinal replacement of hESC RPE in subjects with Stargardt's and GA [2,5] .…”

Section: Rpe Cells Replacementmentioning

confidence: 99%

Stem cell therapy for retinal diseases

Garcia

Mendonça

Brant

et al. 2015

WJSC

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In this review, we discuss about current knowledge about stem cell (SC) therapy in the treatment of retinal degeneration. Both human embryonic stem cell and induced pluripotent stem cell has been growth in culture for a long time, and started to be explored in the treatment of blinding conditions. The Food and Drug Administration, recently, has granted clinical trials using SC retinal therapy to treat complex disorders, as Stargardt's dystrophy, and patients with geographic atrophy, providing good outcomes. This study's intent is to overview the critical regeneration of the subretinal anatomy through retinal pigment epithelium transplantation, with the goal of reestablish important pathways from the retina to the occipital cortex of the brain, as well as the differentiation from pluripotent quiescent SC to adult retina, and its relationship with a primary retinal injury, different techniques of transplantation, management of immune rejection and tumorigenicity, its potential application in improving patients' vision, and, finally, approaching future directions and challenges for the treatment of several conditions.

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“…These cells provide a window for testing the efficacy of gene-or drug-based therapies, elucidate new mechanisms and pathways of disease, and enable researchers to experiment with the parameters for successful cell replacement therapy in vitro. The efforts of the biotechnology industry to make large-scale stem cell production feasible will only make stem cell technology more widely accessible (Borooah et al 2013). Major progress has also been made in developing Good Manufacturing Practice (GMP) laboratories and bringing iPS applications to clinical trials.…”

Section: Future Directionsmentioning

confidence: 99%

Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells

Chan

Nguyen

et al. 2015

Retinal Degenerative Diseases

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Interest in generating human induced pluripotent stem (iPS) cells for stem cell modeling of diseases has overtaken that of patient-specific human embryonic stem cells due to the ethical, technical, and political concerns associated with the latter. In ophthalmology, researchers are currently using iPS cells to explore various applications, including: (1) modeling of retinal diseases using patient-specific iPS cells; (2) autologous transplantation of differentiated retinal cells that undergo gene correction at the iPS cell stage via gene editing tools (e.g., CRISPR/Cas9, TALENs and ZFNs); and (3) autologous transplantation of patient-specific iPS-derived retinal cells treated with gene therapy. In this review, we will discuss the uses of patient-specific iPS cells for differentiating into retinal pigment epithelium (RPE) cells, uncovering disease pathophysiology, and developing new treatments such as gene therapy and cell replacement therapy via autologous transplantation.

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Using human induced pluripotent stem cells to treat retinal disease

Cited by 61 publications

References 165 publications

Let There Be Light: Gene and Cell Therapy for Blindness

Let There Be Light: Gene and Cell Therapy for Blindness

Stem cell therapy for retinal diseases

Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells

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