专题:CRISPR and Genetic Engineering

This cluster of papers focuses on the use of CRISPR-Cas systems for genome engineering, RNA-guided gene editing, and bacterial immunity. It covers topics such as Cas9 nucleases, off-target effects, multiplex genome editing, and genetic screening using CRISPR technology.
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In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia

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Quadruple pegRNA enables programmable and efficient large genomic insertion

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CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease

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In vivo site-specific engineering to reprogram T cells

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In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial

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Bacterial defense mechanisms against bacteriophages: an evolutionary arms race

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Spatial perturb-seq: single-cell functional genomics within intact tissue architecture

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RNA interference-based pesticides: Mechanism, application, and commercialization in sustainable pest management

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Rapid directed evolution guided by protein language models and epistatic interactions

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Commercial Translation of Electrochemical Biosensors: Supply Chain Strategy, Scale-Up Manufacturing, and Regulatory–Quality Considerations

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