Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration

Purpose of Review A major cause of visual disorders is dysfunction and/or loss of the light-sensitive cells of the retina, the photoreceptors. To develop better treatments for patients, we need to understand how inherited retinal disease mutations result in the dysfunction of photoreceptors. New adv...

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Bibliographic Details
Published in:Current stem cell reports 2017-06, Vol.3 (2), p.112-123
Main Authors: Ovando-Roche, Patrick, Georgiadis, Anastasios, Smith, Alexander J., Pearson, Rachael A., Ali, Robin R.
Format: Article
Language:English
Subjects:
Biomedical and Life Sciences
Biomedical Engineering/Biotechnology
Biomedicine
Cell Biology
Cell therapy
CRISPR
Gene Therapy
Genome Editing (SN Waddington and HC O'Neill
Genome Editing (SN Waddington and HC O'Neill, Section Editors)
Genomes
Immunology
Life Sciences
Organoids
Photoreceptors
Pluripotency
Retina
Retinal degeneration
Section Editors
Stem Cells
Topical Collection on Genome Editing
Vision
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Summary:Purpose of Review A major cause of visual disorders is dysfunction and/or loss of the light-sensitive cells of the retina, the photoreceptors. To develop better treatments for patients, we need to understand how inherited retinal disease mutations result in the dysfunction of photoreceptors. New advances in the field of stem cell and gene editing research offer novel ways to model retinal dystrophies in vitro and present opportunities to translate basic biological insights into therapies. This brief review will discuss some of the issues that should be taken into account when carrying out disease modelling and gene editing of retinal cells. We will discuss (i) the use of human induced pluripotent stem cells (iPSCs) for disease modelling and cell therapy; (ii) the importance of using isogenic iPSC lines as controls; (iii) CRISPR/Cas9 gene editing of iPSCs; and (iv) in vivo gene editing using AAV vectors. Recent Findings Ground-breaking advances in differentiation of iPSCs into retinal organoids and methods to derive mature light sensitive photoreceptors from iPSCs. Furthermore, single AAV systems for in vivo gene editing have been developed which makes retinal in vivo gene editing therapy a real prospect. Summary Genome editing is becoming a valuable tool for disease modelling and in vivo gene editing in the retina.
ISSN:2198-7866
2198-7866
DOI:10.1007/s40778-017-0078-4