Stem cells for investigation and treatment of inherited retinal disease

Budd A Tucker; Robert F Mullins; Edwin M Stone

doi:10.1093/hmg/ddu124

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Stem cells for investigation and treatment of inherited retinal disease

Journal article

Open access

Peer reviewed

Stem cells for investigation and treatment of inherited retinal disease

Budd A Tucker, Robert F Mullins and Edwin M Stone

Human molecular genetics, Vol.23(R1), pp.R9-R16

09/15/2014

DOI: 10.1093/hmg/ddu124

PMCID: PMC4170716

PMID: 24647603

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Abstract

Vision is the most important human sense. It facilitates every major activity of daily living ranging from basic communication, mobility and independence to an appreciation of art and nature. Heritable diseases of the retina, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world, collectively affecting as many as one-third of all people over the age of 75, to some degree. For decades, scientists have dreamed of preventing vision loss or of restoring the vision of patients affected with retinal degeneration through some type of drug, gene or cell-based transplantation approach. In this review, we will discuss the current literature pertaining to retinal transplantation. We will focus on the use of induced pluripotent stem cells for interrogation of disease pathophysiology, analysis of drug and gene therapeutics and as a source of autologous cells for cell replacement.

Stem Cell Transplantation

Animals

Macular Degeneration - therapy

Retinitis Pigmentosa - therapy

Humans

Induced Pluripotent Stem Cells - transplantation

Retinal Degeneration - therapy

Clustered Regularly Interspaced Short Palindromic Repeats - genetics

Genome

Disease Models, Animal

Details

Title: Subtitle: Stem cells for investigation and treatment of inherited retinal disease
Creators: Budd A Tucker - Stephen A. Wynn Institute for Vision Research and
Robert F Mullins - Stephen A. Wynn Institute for Vision Research and
Edwin M Stone - Stephen A. Wynn Institute for Vision Research and Howard Hughes Medical Institute, Department of Ophthalmology and Visual Science, Carver College of Medicine, University of Iowa, Iowa city, IA, USA edwin-stone@uiowa.edu
Resource Type: Journal article
Publication Details: Human molecular genetics, Vol.23(R1), pp.R9-R16
DOI: 10.1093/hmg/ddu124
PMID: 24647603
PMCID: PMC4170716
NLM abbreviation: Hum Mol Genet
ISSN: 1460-2083
eISSN: 1460-2083
Publisher: England
Grant note: DP2OD00748301 / NIH HHS DP2 OD007483 / NIH HHS EY016822 / NEI NIH HHS Howard Hughes Medical Institute
Language: English
Date published: 09/15/2014
Academic Unit: Iowa Neuroscience Institute; Ophthalmology and Visual Sciences
Record Identifier: 9983980047102771

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