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Genome Editing of Induced Pluripotent Stem Cells Using CRISPR/Cas9 Ribonucleoprotein Complexes to Model Genetic Ocular Diseases

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Induced Pluripotent Stem Cells and Human Disease

Part of the book series: Methods in Molecular Biology ((MIMB,volume 2549))

Abstract

Genome editing with the use of CRISPR/Cas9 ribonucleoprotein complexes of induced pluripotent stem cells can be used to model many diseases. The combination of stem cells and gene editing technologies is a valuable tool to study ocular disorders, as many have been identified to be caused by specific genetic mutations. This protocol provides experimentally derived guidelines for genome editing of human induced pluripotent stem cells (iPSCs) using CRISPR/Cas9 ribonucleoprotein complexes to generate iPSCs harboring single nucleotide variants from ocular disorders. Edited iPSC can be further differentiated into retinal cells in order to study disease mechanisms as well as screen potential therapies.

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Author information

Authors and Affiliations

  1. The Ocular Genomics Institute at Massachusetts Eye and Ear, Harvard Medical School, Boston, MA, USA

    Heran Getachew, Blanca Chinchilla & Rosario Fernandez-Godino

Authors
  1. Heran Getachew
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  2. Blanca Chinchilla
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  3. Rosario Fernandez-Godino
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Corresponding author

Correspondence to Rosario Fernandez-Godino .

Editor information

Editors and Affiliations

  1. Ottawa, ON, Canada

    Kursad Turksen

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Getachew, H., Chinchilla, B., Fernandez-Godino, R. (2021). Genome Editing of Induced Pluripotent Stem Cells Using CRISPR/Cas9 Ribonucleoprotein Complexes to Model Genetic Ocular Diseases. In: Turksen, K. (eds) Induced Pluripotent Stem Cells and Human Disease. Methods in Molecular Biology, vol 2549. Humana, New York, NY. https://doi.org/10.1007/7651_2021_409

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  • DOI: https://doi.org/10.1007/7651_2021_409

  • Published:

  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-0716-2584-2

  • Online ISBN: 978-1-0716-2585-9

  • eBook Packages: Springer Protocols

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