6 Result(s)
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Article
Open AccessCytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity
Cytosine base editors (CBEs) enable efficient, programmable reversion of T•A to C•G point mutations in the human genome. Recently, cytosine base editors with rAPOBEC1 were reported to induce unguided cytosine ...
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Open AccessPublisher Correction: Pairwise library screen systematically interrogates Staphylococcus aureus Cas9 specificity in human cells
The original HTML version of this Article incorrectly listed an affiliation of Josh Tycko as ‘Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305, USA’, instead of the correct ‘P...
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Article
Open AccessPairwise library screen systematically interrogates Staphylococcus aureus Cas9 specificity in human cells
Therapeutic genome editing with Staphylococcus aureus Cas9 (SaCas9) requires a rigorous understanding of its potential off-target activity in the human genome. Here we report a high-throughput screening approach ...
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Open AccessCharacterization of the interplay between DNA repair and CRISPR/Cas9-induced DNA lesions at an endogenous locus
The CRISPR–Cas9 system provides a versatile toolkit for genome engineering that can introduce various DNA lesions at specific genomic locations. However, a better understanding of the nature of these lesions a...
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Open AccessRecombinant human N-acetylgalactosamine-6-sulfate sulfatase (GALNS) produced in the methylotrophic yeast Pichia pastoris
Mucopolysaccharidosis IV A (MPS IV A, Morquio A disease) is a lysosomal storage disease (LSD) produced by mutations on N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Recently an enzyme replacement therapy ...
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Open AccessContext influences on TALE–DNA binding revealed by quantitative profiling
Transcription activator-like effector (TALE) proteins recognize DNA using a seemingly simple DNA-binding code, which makes them attractive for use in genome engineering technologies that require precise target...
