Enhancement of prime editing via xrRNA motif-joined pegRNA

Article Biotechnology & Applied Microbiology

Engineered pegRNAs improve prime editing efficiency

James W. Nelson et al.

Summary: Prime editing technology enhances editing efficiency by optimizing the structure of pegRNAs, preventing degradation of the 3' region from affecting system activity, without increasing the risk of off-target editing.

NATURE BIOTECHNOLOGY (2022)

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Article Biotechnology & Applied Microbiology

CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells

Karl Petri et al.

Summary: This study demonstrated prime editing using purified ribonucleoprotein complexes, achieving desired edits in zebrafish embryos and human cells. However, unintended insertions, deletions, and guide RNA scaffold incorporations were also observed.

NATURE BIOTECHNOLOGY (2022)

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Article Multidisciplinary Sciences

In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice

Luke W. Koblan et al.

Summary: Using adenine base editor (ABE), researchers successfully corrected the pathogenic mutation in fibroblasts from children with Hutchinson-Gilford progeria syndrome, mitigating RNA mis-splicing, reducing progerin levels, and correcting nuclear abnormalities. In vivo base editing with AAV9 encoding ABE in transgenic mice resulted in substantial, durable correction of the mutation, restoration of normal RNA splicing, and improvement of vascular pathology, ultimately extending the lifespan of the mice. These findings demonstrate the potential of in vivo base editing as a treatment for genetic diseases.

NATURE (2021)

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Article Multidisciplinary Sciences

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

Kiran Musunuru et al.

Summary: This study demonstrates the efficient and precise modification of disease-related genes in living cynomolgus monkeys using CRISPR base editors delivered in vivo via lipid nanoparticles. The results provide proof-of-concept for the potential therapeutic application of CRISPR base editors in making precise single-nucleotide changes in target genes, supporting a 'once-and-done' approach for the treatment of diseases.

NATURE (2021)

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Article Biotechnology & Applied Microbiology

High-efficiency prime editing with optimized, paired pegRNAs in plants

Qiupeng Lin et al.

Summary: Designing prime binding sites with a certain melting temperature and using two pegRNAs can substantially enhance the efficiency of prime editing.

NATURE BIOTECHNOLOGY (2021)

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Article Biotechnology & Applied Microbiology

Genome-wide specificity of prime editors in plants

Shuai Jin et al.

Summary: The study systematically evaluated the mismatch tolerance, off-target editing, and genome-wide variants induced by prime editors in plant cells, showing high precision and low off-target effects in plant genome editing.

NATURE BIOTECHNOLOGY (2021)

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Article Biochemical Research Methods

High-Performance Allosteric Conditional Guide RNAs for Mammalian Cell-Selective Regulation of CRISPR/Cas

Lisa M. Hochrein et al.

Summary: The activity of conditional guide RNA (cgRNA) is dependent on the presence of an RNA trigger, enabling cell-selective regulation of CRISPR/Cas function. Allosteric cgRNA mechanisms allow independent design of target and trigger sequences, providing flexibility to select regulatory targets and scope independently. The high-performance allosteric cgRNAs demonstrated for ON -> OFF and OFF -> ON logic in mammalian cells lay the foundation for programmable cell-selective regulation applications.

ACS SYNTHETIC BIOLOGY (2021)

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Letter Cell Biology

Enhancing prime editing by Csy4-mediated processing of pegRNA

Yao Liu et al.

CELL RESEARCH (2021)

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Article Biochemistry & Molecular Biology

Mechanical strength of RNA knot in Zika virus protects against cellular defenses

Meng Zhao et al.

Summary: Unusual knot-like structures in viral exoribonuclease-resistant RNAs prevent digestion by host RNases to enhance infection. Unfolding a Zika virus xrRNA with optical tweezers revealed it as the most mechanically stable RNA observed, with a knot formed by threading the 5' end into a three-helix junction to generate extreme force resistance. The pseudoknot and tertiary contacts stabilizing the threaded 5' end were essential for RNase resistance.

NATURE CHEMICAL BIOLOGY (2021)

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Article Biotechnology & Applied Microbiology

Targeted mutagenesis in mouse cells and embryos using an enhanced prime editor

Soo-Ji Park et al.

Summary: Prime editors, a novel genome-editing tool, can induce targeted mutations with high efficiency. By utilizing two strategies to improve editing efficiency, we successfully generated Igf2 mutant mice with editing frequencies of up to 47% and observed important phenotypic changes.

GENOME BIOLOGY (2021)

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Article Biotechnology & Applied Microbiology

Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression

Pan Gao et al.

Summary: In mice, both the PE2 and HDR systems led to cell-specific loss of expression of an mRNA/long noncoding RNA gene pair with single nucleotide variants in TFBS. However, PE2 showed higher editing efficiency and lower off-target mutation risk compared to HDR.

GENOME BIOLOGY (2021)

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Article Dermatology