2019
DOI: 10.1111/trf.15126
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Clinical applications of CRISPR‐based genome editing and diagnostics

Abstract: Clustered regularly interspaced short palindromic repeats (CRISPR)‐driven genome editing has rapidly transformed preclinical biomedical research by eliminating the underlying genetic basis of many diseases in model systems and facilitating the study of disease etiology. Translation to the clinic is under way, with announced or impending clinical trials utilizing ex vivo strategies for anticancer immunotherapy or correction of hemoglobinopathies. These exciting applications represent just a fraction of what is … Show more

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Cited by 34 publications
(29 citation statements)
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References 128 publications
(287 reference statements)
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“…Genome editing, a powerful toolset involving deliberate changes in the DNA sequence, is leading to a new era of molecular medicine against cancer, genetic disorders and beyond [ 1 ]. Editing methods currently available include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR).…”
Section: Introductionmentioning
confidence: 99%
“…Genome editing, a powerful toolset involving deliberate changes in the DNA sequence, is leading to a new era of molecular medicine against cancer, genetic disorders and beyond [ 1 ]. Editing methods currently available include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR).…”
Section: Introductionmentioning
confidence: 99%
“…A single guide RNA (sgRNA) directs the endonuclease Cas9 to DNA sequence which has been targeted, and initiates site-specific manipulation [79]. The Type II CRISPR/Cas9 system is an extensively used DNA-editing method, as a result of the ability to design CRISPR-guided nucleases in this system easily and relatively quickly [20,27].…”
Section: Bacterial Diagnosis By Crispr Systemmentioning
confidence: 99%
“…Examples of target sequences include oncogenic mutation sequences or viral and bacterial sequences derived from the infectious agent. The goal of CRISPR systems is to identify the specific pathogens, as well as to repair alleles that cause disease through specific DNA sequence editing at exact locations on the chromosome [ 20 ]. The goal of CRISPR systems is to identify the specific pathogens, as well as to repair alleles that cause disease through specific DNA sequence editing at exact locations on the chromosome [ 79 ].…”
Section: Crispr As a Diagnostic Toolmentioning
confidence: 99%
“…Such ex vivo genome editing is currently the most technically feasible approach, and has the potential to treat devastating blood disorders like sickle cell disease and β-thalassemia. The ex vivo strategy also underlies cancer immunotherapies [66].…”
Section: The Capacity Of Crispr Diagnosticsmentioning
confidence: 99%