2020
DOI: 10.1042/bsr20200127
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Gene editing and CRISPR in the clinic: current and future perspectives

Abstract: Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly interspaced short palindromic repeat DNA sequences)/Cas9 are rapidly progressing into clinical trials. Most clinical use of CRISPR to date has focused on ex vivo gene editing of cells followed by their re-introduction back into the patient. The ex vivo editing approach is highly effective for many disease states, including cancers and s… Show more

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Cited by 146 publications
(121 citation statements)
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References 297 publications
(431 reference statements)
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“…2 Not only have these systems been rapidly deployed as research tools in studying all domains of life from bacteria to humans, but they have offered new routes to advanced therapies for many diseases, from engineered cell lines such as CAR-T cells to editing humans genomes, offering the potential of cures for numerous genetic diseases. [3][4][5][6] CRISPR/Cas systems and S. pyogenes (Cas9) in particular (the most well studied) work remarkably well across a diversity of species and applications and are easier to use than the tools previously available. 7 However, challenges remain in the use of CRISPR/Cas systems, even with S. pyogenes Cas9.…”
Section: Introductionmentioning
confidence: 99%
“…2 Not only have these systems been rapidly deployed as research tools in studying all domains of life from bacteria to humans, but they have offered new routes to advanced therapies for many diseases, from engineered cell lines such as CAR-T cells to editing humans genomes, offering the potential of cures for numerous genetic diseases. [3][4][5][6] CRISPR/Cas systems and S. pyogenes (Cas9) in particular (the most well studied) work remarkably well across a diversity of species and applications and are easier to use than the tools previously available. 7 However, challenges remain in the use of CRISPR/Cas systems, even with S. pyogenes Cas9.…”
Section: Introductionmentioning
confidence: 99%
“…Precise detection of off-target activity is crucial if CRISPR technology is to be used more widely and especially in a clinical setting 78 . However, many existing methods have differing sensitivities 79 making comparisons between studies difficult (e.g.…”
Section: Challenges Inconsistent Off-target Detection Methodsmentioning
confidence: 99%
“…Multifunctional AuNPs carrying TNAs and/or gene editing molecular tools have been mostly evaluated in in vitro models, but several of these concepts have also been proven in vivo. However, it must be said that most of these concepts do not make into the systemic evaluation required for further clinical development and translation, mostly due to hurdles relating to scale-up of production and precise physical characterization of these nanoconjugates, and for the complexity of assessing chronic and whole organism's nanotoxicology [152,153].…”
Section: Translation To the Clinicmentioning
confidence: 99%