Human Retinal Organoids in Therapeutic Discovery: A Review of Applications

Cheng, Lin; Kuehn, Markus H.

doi:10.1007/164_2023_691

Cited by 2 publications

(2 citation statements)

References 128 publications

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“…The utilization of human retinal organoids derived from human induced pluripotent stem cells has enabled the examination of both retinal development and the study of retinal diseases 53–58 . They consist of three nuclear layers and two synaptic layers, replicating the cellular organization observed in the adult retina, and that they exhibit photoreceptor responsiveness to light stimuli and facilitate synaptic transmission of visual information, resulting in light responses in second or third order retinal cells 56 .…”

Section: Discussionmentioning

confidence: 99%

PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F

Ivanchenko,

Hathaway,

Mulhall

et al. 2023

Preprint

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Usher syndrome type 1F (USH1F), resulting from mutations in the protocadherin-15 (PCDH15) gene, is characterized by congenital lack of hearing and balance, and progressive blindness in the form of retinitis pigmentosa. In this study, we explore a novel approach for USH1F gene therapy, exceeding the single AAV packaging limit by employing a dual adeno-associated virus (AAV) strategy to deliver the full-length PCDH15 coding sequence. We demonstrate the efficacy of this strategy in mouse USH1F models, effectively restoring hearing and balance in these mice. Importantly, our approach also proves successful in expressing PCDH15 in clinically relevant retinal models, including human retinal organoids and non-human primate retina, showing efficient targeting of photoreceptors and proper protein expression in the calyceal processes. This research represents a major step toward advancing gene therapy for USH1F and the multiple challenges of hearing, balance, and vision impairment.

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Section: Discussionmentioning

confidence: 99%

PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F

Ivanchenko,

Hathaway,

Mulhall

et al. 2023

Preprint

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show abstract

“…There are two main approaches to establishing ROs (Cheng and Kuehn, 2023 ) and many approaches for generating different regions of BOs (Mayhew and Singhania, 2023 ). The modification and improvement of these methods are continuously ongoing.…”

mentioning

confidence: 99%

Editorial: Stem cell-derived retinal and brain organoid culture for disease modeling

Cheng,

Caldwell,

Cho

et al. 2024

Front. Cell. Neurosci.

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Human Retinal Organoids in Therapeutic Discovery: A Review of Applications

Cited by 2 publications

References 128 publications

PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F

PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F

Editorial: Stem cell-derived retinal and brain organoid culture for disease modeling

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