gene editing

All-in-One CRISPR Genome Editing: A Method for Homology Directed Repair-Based Gene Knock-In in Cultured Cells Using CRISPR-Cas9 System

Between Speculation and Regulation: Science Fiction as a Tool for Legal Inquiry

Abstract This article develops a conceptual framework for using science fiction as a tool for anticipatory legal analysis of emerging technologies. As technologies such as artificial intelligence, gene editing, advanced reproductive systems, and autonomous weaponry advance faster than conventional legal methodologies c...

Glycosomal ABC transporter 3 (GAT3) is associated with antileishmanial drug susceptibility in Leishmania infantum.

It is demonstrated that GAT3 is a non-essential glycosomal ABC transporter that contributes to antileishmanial drug susceptibility in L. infantum and the association between GAT3 deletion and reduced expression of AQP1 and MT suggests a previously unrecognized link between glycosomal transport processes and pathways in...

Modeling Prostate Cancer in Genetically-engineered Mouse Models: A CRISPR/Cas9-mediated Localized Gene Editing Technique in Mouse Anterior Prostate Lobe Cells

Combating New Delhi metallo-beta-lactamase-1 (NDM-1): an integrated review of inhibitor development and next-generation therapeutic platforms

This review provides details on the known inhibitor classes, including thiol-based compounds, hydroxamates, bicyclic boronates, repurposed drugs, and natural products, and discusses the challenges related to pharmacokinetics, toxicity, and variant-specific effectiveness.

Gene editing of the transmembrane kinase SOAK1 fine-tunes reactive oxygen species production for enhanced disease resistance in potato.

An RLCK-V subfamily member in potato is reported, StSOAK1, which negatively regulates disease resistance to the oomycete Phytophthora infestans and the fungus Botrytis cinerea and reveals a regulatory mechanism of potato StRBOHB, which occurs in parallel with its paradigmatic AtPBL13-mediated regulation in Arabidopsis.

Establishment of a CRISPR‐Cas9 Library for Indica Rice and Identification of OsOPR5 (LOC_Os06g11210) as a Regulator of Root Architecture

This study establishes an efficient genome‐editing platform for indica rice and identifies OsOPR5 as a novel regulator of root development, a member of the 12‐oxophytodienoate reductase family in root architecture.

Porcine kidney xenotransplantation as a bridge to allotransplantation: a first-in-human study.

This case shows that porcine kidney xenotransplantation can provide prolonged renal support and be discontinued without clinically significant allosensitisation or zoonotic infection.

In Vivo Base Editing Partially Rescues Bone Dysplasia in a Mouse Model of Hutchinson‐Gilford Progeria Syndrome

In vivo delivery of a locus‐specific DNA base editor to bone tissue is demonstrated, the timing of treatment required for maximum efficacy is delineated, and it is suggested that this system might be tailored for application to other monogenic bone disorders.

Hemoglobinopathies and Cellular Therapy: What Pediatricians Need to Know About Indications and Counseling.

The pathophysiology, diagnosis, management, and curative options for SCD and transfusion-dependent thalassemia are summarized, emphasizing allogeneic hematopoietic stem cell transplantation and approved gene therapies.

Een nieuwe rol voor VPS13C bij de regulatie van mTORC1-signalering op contactpunten tussen plasmamembraan en endoplasmatisch reticulum.

Dementia is one of the main afflictions of today's society, with Dementia with Lewy Bodies (DLB) representing around 25% of all dementias, only second to Alzheimer's Disease. DLB, together with Parkinson's Disease (PD), comprise Lewy Body Diseases (LBD), a group of disorders characterized by the presence of Lewy bodies...

EukaUTR: a foundation model for functional modelling and design of eukaryotic 3′ UTRs

Eukaryotic mRNA 3′ UTRs encode regulatory signals that shape post-transcriptional regulation, RNA fate and gene expression. However, a foundation model that spans broad eukaryotic 3′ UTR sequence space while unifying prediction, interpretation and design within a single framework is still lacking. Here we present EukaU...