Cas9 as a versatile tool for engineering biology
Open accessAbstract
RNA-guided Cas9 nucleases derived from clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems have dramatically transformed our ability to edit the genomes of diverse organisms. We believe tools and techniques based on Cas9, a single unifying factor capable of colocalizing RNA, DNA and protein, will grant unprecedented control over cellular organization, regulation and behavior. Here we describe the Cas9 targeting methodology, detail current and prospective engineering advances and suggest potential applications ranging from basic science to the clinic.
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Engineered nucleases including ZFN, TALEN, and CRISPR Cas9 can disrupt target genes and have raised prospects of selectively deleting HIV-1 proviral DNA integrated into the host genome.
“Advances in the engineered nucleases including zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR) associated 9 (Cas9) that can disrupt target genes have raised prospects of selectively deleting HIV-1 proviral DNA integrated into the host genome”
From RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection (2014) · cites this paper as [10]
If HIV-1 mutations confer resistance to one Cas9/gRNA-based therapy, HIV-1 variants can be genotyped to enable another personalized therapy for individual patients.
“even if HIV-1 mutations confer resistance to one Cas9/gRNA-based therapy, as described above, HIV-1 variants can be genotyped to enable another personalized therapy for individual patients (10).”
From RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection (2014) · cites this paper as [10]
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- Authors
- Mali, P., Esvelt, K. M., & Church, G. M.
- Affiliations
- Harvard University (US)
- Corresponding author
- Mali, Prashant
- Esvelt, Kevin M
- Venue
- Nature Methods
- Published
- 2013-09-27
- Volume
- 10
- Issue
- 10
- Pages
- 957-963
- ISSN
- 1548-7091 (ISSN-L)
- 1548-7105
- PMC
- PMC4051438
- OpenAlex
- W2089589904
- Cited by
- 1,197
- References
- 76
- Citations by year
- 2026: 19 · 2025: 36 · 2024: 50 · 2023: 60 · 2022: 70 · 2021: 78 · 2020: 78 · 2019: 109 · 2018: 99 · 2017: 124 · 2016: 158 · 2015: 152 · 2014: 158 · 2013: 3
- FWCI
- 14.75
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Methods (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Digital Access to Scholarship at Harvard (DASH) (Harvard University) (acceptedVersion)
- Funders
- National Human Genome Research Institute (P50HG005550)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Bacterial Genetics and Biotechnology
- Keywords
- CRISPR/Cas9, genome editing, RNA-guided nucleases, engineering biology, CRISPR, gene expression regulation, therapeutic applications
- MeSH terms
- Genetic Engineering, Repetitive Sequences, Nucleic Acid, Ribonucleases, Streptococcus pyogenes, Genome, Genome, Bacterial, RNA Editing, DNA Cleavage, Bioengineering, RNA, Small Untranslated, Clustered Regularly Interspaced Short Palindromic Repeats
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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