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New Perspectives in Stem Cell Transplantation and Associated Therapies to Treat Retinal Diseases: From Gene Editing to 3D Bioprinting

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Abstract

Inherited and non-inherited retinopathies can affect distinct cell types, leading to progressive cell death and visual loss. In the last years, new approaches have indicated exciting opportunities to treat retinopathies. Cell therapy in retinitis pigmentosa, age-related macular disease, and glaucoma have yielded encouraging results in rodents and humans. The first two diseases mainly impact the photoreceptors and the retinal pigmented epithelium, while glaucoma primarily affects the ganglion cell layer. Induced pluripotent stem cells and multipotent stem cells can be differentiated in vitro to obtain specific cell types for use in transplant as well as to assess the impact of candidate molecules aimed at treating retinal degeneration. Moreover, stem cell therapy is presented in combination with newly developed methods, such as gene editing, Müller cells dedifferentiation, sheet & drug delivery, virus-like particles, optogenetics, and 3D bioprinting. This review describes the recent advances in this field, by presenting an updated panel based on cell transplants and related therapies to treat retinopathies.

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Funding

This research was supported by grants from Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP, Brazil, #2019/17892-8 and #2020/11667-0), Conselho Nacional de Desenvolvimento Científico e Tecnológico (CNPq, Brazil. #312047/2017-7 and #315372/2021-4), Coordenação Aperfeiçoamento de Pessoal de Nível Superior (CAPES, Brazil, financial code 001). The following authors were recipients of fellowships from FAPESP: GBS (#2021/03711-1 and #2021/14227-3), THLV (#2020/02035-0 and #2021/11969-9), MIM (#2017/26388-6).

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Authors and Affiliations

  1. Laboratório de Neurogenética, Universidade Federal do ABC, São Bernardo do Campo, Santo André, SP, Brazil

    Gabrieli Bovi dos Santos, Théo Henrique de Lima-Vasconcellos, Marília Inês Móvio & Alexandre Hiroaki Kihara

  2. Department of Dermatology, Medical Sciences Center, University of Wisconsin-Madison, Rm 4385, 1300 University Avenue, Madison, WI, 53706, USA

    Alexander Birbrair

  3. Departamento de Biologia Celular e do Desenvolvimento, Instituto de Ciências Biomédicas, Universidade de São Paulo USP, São Paulo, SP, Brazil

    Carolina Beltrame Del Debbio

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  1. Gabrieli Bovi dos Santos
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GBS, THLV, MIM, and AHK conceived the review and wrote the first draft. AHK, AB, and CBDD revised the manuscript. All authors read and approved the final manuscript.

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Correspondence to Alexandre Hiroaki Kihara.

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Gabrieli Bovi dos Santos and Théo Henrique de Lima-Vasconcellos contributed equally to this work.

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Bovi dos Santos, G., de Lima-Vasconcellos, T.H., Móvio, M.I. et al. New Perspectives in Stem Cell Transplantation and Associated Therapies to Treat Retinal Diseases: From Gene Editing to 3D Bioprinting. Stem Cell Rev and Rep 20, 722–737 (2024). https://doi.org/10.1007/s12015-024-10689-4

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