Abstract
Despite the unparalleled therapeutic promise of genome editing, its curative power is currently limited by the substantial difficulty in delivering DNA-cutting enzymes to the cells in need of correction. A recent study demonstrates the potential for the delivery of pre-assembled genome-editing enzymes in the form of ribonucleoprotein complexes, which were used to rescue a mouse model of fragile X syndrome (FXS).
Copyright © 2018 Elsevier Ltd. All rights reserved.
MeSH terms
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Animals
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Biological Transport
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Brain / metabolism
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Brain / pathology
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CRISPR-Associated Protein 9 / genetics*
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CRISPR-Associated Protein 9 / metabolism
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CRISPR-Cas Systems*
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Clustered Regularly Interspaced Short Palindromic Repeats
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Dependovirus / genetics
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Dependovirus / metabolism
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Disease Models, Animal
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Fragile X Mental Retardation Protein / genetics
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Fragile X Mental Retardation Protein / metabolism
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Fragile X Syndrome / genetics
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Fragile X Syndrome / metabolism
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Fragile X Syndrome / pathology
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Fragile X Syndrome / therapy*
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Gene Deletion
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Gene Editing / methods*
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Gene Transfer Techniques*
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Gold / administration & dosage
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Gold / chemistry
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Gold / metabolism
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Humans
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Metal Nanoparticles / administration & dosage
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Metal Nanoparticles / chemistry
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Mice
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Mice, Knockout
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Ribonucleoproteins / genetics*
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Ribonucleoproteins / metabolism
Substances
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Fmr1 protein, mouse
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Ribonucleoproteins
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Fragile X Mental Retardation Protein
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Gold
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CRISPR-Associated Protein 9