Induced Pluripotent Stem Cells for Inherited Optic Neuropathies-Disease Modeling and Therapeutic Development


iPSCs offer an elegant, patient-focused solution to the investigation of the genetic defects and disease mechanisms underpinning IONs. Furthermore, this group of disorders is uniquely amenable to both the disease modeling capability and the therapeutic potential that iPSCs offer. This fast-moving area will remain at the forefront of both basic and translational ION research in the coming years, with the potential to accelerate the development of effective therapies for patients affected with these blinding diseases. Copyright © 2021 by North American Neuro-Ophthalmology Society.

Authors Harvey JP, Sladen PE, Yu-Wai-Man P, Cheetham ME
Journal Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society
Publication Date 2022 Mar 1;42(1):35-44
PubMed 34629400
DOI 10.1097/wno.0000000000001375

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