Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies

Retinal diseases constitute a genetically and phenotypically diverse group of clinical conditions leading to vision impairment or blindness with limited treatment options. Advances in reprogramming of somatic cells to induced pluripotent stem cells and generation of three‐dimensional organoids resem...

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Vydané v:Stem cells (Dayton, Ohio) Ročník 38; číslo 10; s. 1206 - 1215
Hlavní autori: Kruczek, Kamil, Swaroop, Anand
Médium: Journal Article
Jazyk:English
Vydavateľské údaje: Hoboken, USA John Wiley & Sons, Inc 01.10.2020
Oxford University Press
Predmet:
Animals
Blindness
Cell Differentiation
Concise Review
Drug therapy
Electrophysiology
Genetic Therapy
Humans
Models, Biological
neural differentiation
Neurodegeneration
Organoids
Organoids - pathology
Pluripotency
Pluripotent Stem Cells - cytology
Regenerative Medicine
Retina
Retina - pathology
Retinal cells
retinal photoreceptors
Somatic cells
somatic stem cells
Stem cells
retinal photoreceptors
neural differentiation
somatic stem cells
retina
ISSN:1066-5099, 1549-4918, 1549-4918
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Popis
Shrnutí:Retinal diseases constitute a genetically and phenotypically diverse group of clinical conditions leading to vision impairment or blindness with limited treatment options. Advances in reprogramming of somatic cells to induced pluripotent stem cells and generation of three‐dimensional organoids resembling the native retina offer promising tools to interrogate disease mechanisms and evaluate potential therapies for currently incurable retinal neurodegeneration. Next‐generation sequencing, single‐cell analysis, advanced electrophysiology, and high‐throughput screening approaches are expected to greatly expand the utility of stem cell‐derived retinal cells and organoids for developing personalized treatments. In this review, we discuss the current status and future potential of combining retinal organoids as human models with recent technologies to advance the development of gene, cell, and drug therapies for retinopathies. Retinal organoids offer a unique human model system to study mechanisms of disease pathogenesis and for developing therapies.
Bibliografia:Funding information
National Eye Institute, Grant/Award Numbers: ZIAEY000546, ZIAEY000474, ZIAEY000450
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Funding information National Eye Institute, Grant/Award Numbers: ZIAEY000546, ZIAEY000474, ZIAEY000450
ISSN:1066-5099
1549-4918
1549-4918
DOI:10.1002/stem.3239