Pengpeng Liu

Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo

The paper develops fully modified SpyCas9 guide RNAs that maintain or enhance genome editing efficacy in vitro and in vivo.

Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery

SORT LNPs enable efficient Cas9-mediated repair of a pathogenic allele in skeletal muscle in a mouse model of LGMDR7.

Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells

This study shows that gene editing of HSCs without ex vivo culture reduces genotoxicity while maintaining therapeutic efficacy for beta-hemoglobinopathy treatment.

A brown fat-enriched adipokine, ASRA, is a leptin receptor antagonist that stimulates appetite

This study identifies ASRA as a leptin receptor antagonist that stimulates appetite and contributes to obesity by counteracting leptin signaling.

Enhancing Prime Editing Efficiency and Flexibility with Tethered and Split pegRNAs

This study improves prime editing efficiency and flexibility by introducing tethered and split pegRNAs, achieving 2-4-fold higher mutation rates in cell lines.

Adenine Base Editing in vivo with a Single Adeno-Associated Virus Vector

This study develops a compact adenine base editor (Nme2-ABE) that can be delivered via a single AAV vector to efficiently edit mouse genomic loci and revert disease mutations in a tyrosinemia model.

Optimization of NLS Composition Improves CRISPR-Cas12a Editing Rates in Human Primary Cells

This study optimizes the nuclear localization signal (NLS) of Cas12a to enhance editing efficiency in human primary cells without compromising specificity.

5' Modifications Improve Potency and Efficacy of DNA Donors for Precision Genome Editing

This study shows that 5' modifications to DNA repair templates enhance precision genome editing efficiency in multiple animal models and human cells.

Development of a flexible split prime editor using truncated reverse transcriptase

This study develops a compact prime editor with reduced reverse transcriptase activity, showing efficient editing and enabling flexible split-PE design for in vivo delivery.

UPRmt scales mitochondrial network expansion with protein synthesis via mitochondrial import

The paper shows that mitochondrial network expansion during development is controlled by the UPRmt pathway, which is influenced by protein synthesis and the mitochondrial import capacity of ATFS-1.

GS-Preprocess: Containerized GUIDE-seq Data Analysis Tools with Diverse Sequencer Compatibility

GS-Preprocess simplifies GUIDE-seq analysis by converting standard Illumina sequencer output into compatible input for off-target profiling, enhancing accessibility and reducing costs.

Efficient Homology-directed Repair with Circular ssDNA Donors

This paper demonstrates that circular ssDNA donors improve HDR efficiency and reduce off-target integration compared to linear ssDNA donors in mammalian cells.

NmeCas9 is an intrinsically high-fidelity genome editing platform

The paper demonstrates that NmeCas9, a Cas9 ortholog, exhibits high-fidelity genome editing in human cells with minimal off-target effects and multiple PAM compatibility.

Potent Cas9 inhibition in bacterial and human cells by new anti-CRISPR protein families

The paper identifies and characterizes two new anti-CRISPR protein families that inhibit type II-C Cas9 systems, showing potent inhibition of NmeCas9 and cross-species activity.