gene editing

Nigral DJ-1 gene knockout in tree shrews can induce phosphorylated α-synuclein aggregation and all key phenotypes of Parkinson’s disease

In vivo evidence is provided supporting a model in which DJ-1 deficiency can promote key PD-like phenotypes in tree shrews, potentially involving PSer129αSyn accumulation, nigral dopaminergic neuron loss, and motor dysfunction.

The Evolution of Crucial Vertebrate Muscle Proteins

Supplemental data failes to accompany the dissertation titled "The Evolution of Crucial Verterbate Muscle Proteins." Descriptions of files are provided below. Data S1: Myosin expression values and sample metadata from publicly available datasets. The file does not contain Lonchura striata expression data. NCBI accessio...

The Evolution of Crucial Vertebrate Muscle Proteins

Supplemental data failes to accompany the dissertation titled "The Evolution of Crucial Verterbate Muscle Proteins." Descriptions of files are provided below. Data S1: Myosin expression values and sample metadata from publicly available datasets. The file does not contain Lonchura striata expression data. NCBI accessio...

CRISPR-mediated excision of HTLV-1 reduces proviral loads in PBMCs from HAM/TSP patients

It is demonstrated in vitro that CRISPR can be used to excise the HTLV-1 genome and reduce proviral loads in PBMCs from HAM/TSP patients and may serve as a platform for curing HAM/TSP.

Precise genomic integration of large DNA fragments by donor-directed annealing using prime editing.

A programmable gene replacement tool, named prime assembly (PA), which adapts prime editors to produce one or two pairs of 3'-flaps on both the genome and donor DNA, allowing megabase-scale genomic excision and/or kilobase-scale donor insertion at the gene of interest.

Phytomelatonin-mediated epigenetic and RNA regulatory networks in plant abiotic stress resilience.

This work proposes that phytomelatonin functions as an epigenetic and epitranscriptomic trigger capable of converting transient stress perception into durable transcriptional competence and outlines how single-cell multi-omics, targeted epigenome editing, epitranscriptomic profiling and field-scale validation of primin...

The emerging roles of non-coding RNAs in cancer.

This review examines how different types of ncRNAs contribute to cancer initiation, progression, and treatment resistance, and assesses their potential as diagnostic markers, prognostic factors, and therapeutic targets.

Functional assessment of inherited myeloid neoplasm-associated SAMD9L germline variants via Monoallelic CRISPR modelling.

Functional evidence is provided to fine-tune the classification of these SAMD9L variants and significantly advance the understanding of the molecular mechanisms by which SAMD9L variants drive inherited myeloid neoplasms.

Targeting Cas9 to Perform Rescue Experiments in Stably Transduced Knockout HEK293 Cells.

Cas9 ablation enabled restoration of CUL4B expression and function following introduction of a CUL4B expression plasmid, providing proof of concept for a broadly applicable approach to studying protein function through rescue experiments.

Advancing variant-agnostic liver-directed gene editing using homology-independent targeted integration.

A PepFect14 analog improves non‐viral CRISPR delivery in primary human cells to facilitate genome editing and repair

A PepFect14 analog is employed to deliver high-fidelity Cas9-ribonucleoproteins and non-viral repair templates into primary human skin cells to mediate gene editing and repair targeting genes underlying the group of genetic skin blistering disorders epidermolysis bullosa (EB).

Gene knockout of P450s CYP392A16 and CYP392D2 reveals unexpected in vivo effects and transcriptional rewiring in the extreme generalist Tetranychus urticae.

It is demonstrated that even resistance-associated detoxification genes that are considered functionally validated in vitro, can have strong context-dependent phenotypic effects in vivo, underscoring the complexity of resistance gene networks and highlighting the need to interpret CRISPR/Cas9 knockout phenotypes within...