Genome editing for blood disorders: state of the art and recent advances Article Swipe
Marianna Romito
,
Rajeev Rai
,
Adrian J. Thrasher
,
Alessia Cavazza
·
YOU?
·
· 2019
· Open Access
·
· DOI: https://doi.org/10.1042/etls20180147
YOU?
·
· 2019
· Open Access
·
· DOI: https://doi.org/10.1042/etls20180147
In recent years, tremendous advances have been made in the use of gene editing to precisely engineer the genome. This technology relies on the activity of a wide range of nuclease platforms — such as zinc-finger nucleases, transcription activator-like effector nucleases, and the CRISPR–Cas system — that can cleave and repair specific DNA regions, providing a unique and flexible tool to study gene function and correct disease-causing mutations. Preclinical studies using gene editing to tackle genetic and infectious diseases have highlighted the therapeutic potential of this technology. This review summarizes the progresses made towards the development of gene editing tools for the treatment of haematological disorders and the hurdles that need to be overcome to achieve clinical success.
Related Topics
Concepts
Genome editing
Transcription activator-like effector nuclease
Zinc finger nuclease
CRISPR
Computational biology
Biology
Effector
Cas9
Genome
Gene
Nuclease
Genetics
Immunology
Metadata
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.1042/etls20180147
- https://portlandpress.com/emergtoplifesci/article-pdf/3/3/289/851039/etls-2018-0147c.pdf
- OA Status
- hybrid
- Cited By
- 6
- References
- 94
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W2937307843
All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
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https://openalex.org/W2937307843Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.1042/etls20180147Digital Object Identifier
- Title
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Genome editing for blood disorders: state of the art and recent advancesWork title
- Type
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articleOpenAlex work type
- Language
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enPrimary language
- Publication year
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2019Year of publication
- Publication date
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2019-04-09Full publication date if available
- Authors
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Marianna Romito, Rajeev Rai, Adrian J. Thrasher, Alessia CavazzaList of authors in order
- Landing page
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https://doi.org/10.1042/etls20180147Publisher landing page
- PDF URL
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https://portlandpress.com/emergtoplifesci/article-pdf/3/3/289/851039/etls-2018-0147c.pdfDirect link to full text PDF
- Open access
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YesWhether a free full text is available
- OA status
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hybridOpen access status per OpenAlex
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https://portlandpress.com/emergtoplifesci/article-pdf/3/3/289/851039/etls-2018-0147c.pdfDirect OA link when available
- Concepts
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Genome editing, Transcription activator-like effector nuclease, Zinc finger nuclease, CRISPR, Computational biology, Biology, Effector, Cas9, Genome, Gene, Nuclease, Genetics, ImmunologyTop concepts (fields/topics) attached by OpenAlex
- Cited by
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6Total citation count in OpenAlex
- Citations by year (recent)
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2024: 1, 2022: 2, 2021: 1, 2020: 1, 2019: 1Per-year citation counts (last 5 years)
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94Number of works referenced by this work
- Related works (count)
-
10Other works algorithmically related by OpenAlex
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