Background: Ribozymes are small catalytic RNAs that possess the dual functions of sequence- specific RNA recognition and site- specificcleavage.
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A second mechanism, which is operative at late times during viral replication, involves the site- specificcleavage of primary RNAs.
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Hammerhead ribozymes are effective modulators of gene expression due to their simple structure, site- specificcleavage activity and catalytic potential.
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V(D)J recombination is a highly regulated process, proceeding from a site- specificcleavage to an imprecise end joining.
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V(D)J recombination proceeds from a site- specificcleavage to an imprecise end joining, via generation and resolution of recombination ends.
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Selective inhibitors were applied to dissect the roles of the individual peptidases and to determine the peptidase- specificcleavage map of the hemoglobin molecule.
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Analysis of aggrecanase specificcleavage patterns of a crude cartilage proteoglycan extract were used to validate some of the differences in peptide intensity identified.
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Here, we review DSB repair mechanisms and site- specificcleavage systems that have provided insight into these mechanisms and led to the current gene editing revolution.
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Results: Following AAV-mediated delivery of editing reagents to patient-derived primary human hepatocytes in vivo, OTC locus- specificcleavage was achieved at efficiencies of up to 72%.