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CRISPR/Cas9 Genome Engineering in Human Pluripotent Stem Cells for Modeling of Neurological Disorders

Canals, Isaac LU and Ahlenius, Henrik LU (2021) In Methods in Molecular Biology 2352. p.237-251
Abstract

Recent advances in genome editing have brought new hopes for personalized and precision medicine but have also dramatically facilitated disease modeling studies. Combined with reprogramming approaches, stem cells and differentiation toward neural lineages, genome engineering holds great potential for regenerative approaches and to model neurological disorders. The use of patient-specific induced pluripotent stem cells combined with neural differentiation allows studying the effect of specific mutations in different brain cells. New genome editing tools such as CRISPR/Cas9 represent a step further by facilitating the introduction or correction of specific mutations within the same cell line, thus eliminating variability due to... (More)

Recent advances in genome editing have brought new hopes for personalized and precision medicine but have also dramatically facilitated disease modeling studies. Combined with reprogramming approaches, stem cells and differentiation toward neural lineages, genome engineering holds great potential for regenerative approaches and to model neurological disorders. The use of patient-specific induced pluripotent stem cells combined with neural differentiation allows studying the effect of specific mutations in different brain cells. New genome editing tools such as CRISPR/Cas9 represent a step further by facilitating the introduction or correction of specific mutations within the same cell line, thus eliminating variability due to differences in the genetic background. Here, we present a step-by-step protocol from design to generation of human pluripotent stem cell lines with specific mutations introduced or corrected with CRISPR/Cas9 gene editing that can be used in combination with transcription factor-based protocols to dissect underlying mechanisms of neurological disorders.

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Please use this url to cite or link to this publication:
author
and
organization
publishing date
type
Chapter in Book/Report/Conference proceeding
publication status
published
subject
keywords
CRISPR/Cas9, Disease modeling, Genome editing, Human pluripotent stem cells
host publication
Neural Reprogramming : Methods and Protocols - Methods and Protocols
series title
Methods in Molecular Biology
volume
2352
pages
15 pages
publisher
Humana Press
external identifiers
  • scopus:85111884250
  • pmid:34324191
ISSN
1940-6029
1064-3745
ISBN
978-1-0716-1601-7
978-1-0716-1600-0
DOI
10.1007/978-1-0716-1601-7_16
language
English
LU publication?
yes
additional info
Publisher Copyright: © 2021, Springer Science+Business Media, LLC, part of Springer Nature.
id
df5f76c8-8417-4d1a-ad65-507b576ae9b8
date added to LUP
2021-12-10 09:37:26
date last changed
2024-04-20 17:30:51
@inbook{df5f76c8-8417-4d1a-ad65-507b576ae9b8,
  abstract     = {{<p>Recent advances in genome editing have brought new hopes for personalized and precision medicine but have also dramatically facilitated disease modeling studies. Combined with reprogramming approaches, stem cells and differentiation toward neural lineages, genome engineering holds great potential for regenerative approaches and to model neurological disorders. The use of patient-specific induced pluripotent stem cells combined with neural differentiation allows studying the effect of specific mutations in different brain cells. New genome editing tools such as CRISPR/Cas9 represent a step further by facilitating the introduction or correction of specific mutations within the same cell line, thus eliminating variability due to differences in the genetic background. Here, we present a step-by-step protocol from design to generation of human pluripotent stem cell lines with specific mutations introduced or corrected with CRISPR/Cas9 gene editing that can be used in combination with transcription factor-based protocols to dissect underlying mechanisms of neurological disorders.</p>}},
  author       = {{Canals, Isaac and Ahlenius, Henrik}},
  booktitle    = {{Neural Reprogramming : Methods and Protocols}},
  isbn         = {{978-1-0716-1601-7}},
  issn         = {{1940-6029}},
  keywords     = {{CRISPR/Cas9; Disease modeling; Genome editing; Human pluripotent stem cells}},
  language     = {{eng}},
  pages        = {{237--251}},
  publisher    = {{Humana Press}},
  series       = {{Methods in Molecular Biology}},
  title        = {{CRISPR/Cas9 Genome Engineering in Human Pluripotent Stem Cells for Modeling of Neurological Disorders}},
  url          = {{http://dx.doi.org/10.1007/978-1-0716-1601-7_16}},
  doi          = {{10.1007/978-1-0716-1601-7_16}},
  volume       = {{2352}},
  year         = {{2021}},
}