CRISPR/Cas9 Genome Engineering in Human Pluripotent Stem Cells for Modeling of Neurological Disorders
(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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- author
- Canals, Isaac LU and Ahlenius, Henrik LU
- organization
- publishing date
- 2021
- 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
- 1064-3745
- 1940-6029
- 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
- 2024-09-22 07:25:36
@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 = {{1064-3745}}, 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}}, }