gene editing

Development and optimization of a cell cycle molecular clock based on the ABE-CRISPR system.

The adenine base editor template number (ATN) system is developed, which implements ordered, stepwise editing of double-stranded DNA through a programmable cascade of modifications and enables temporally regulated manipulation of cell fate.

From iPSC Disease Modeling to Epigenome Editing: Toward Therapeutic Reversal of Genomic Imprinting Disorders.

How iPSC-based disease modeling is evolving from a platform for mechanism discovery toward a test bed for therapeutic epigenome engineering is discussed and the major challenges that must be addressed are outlined before epigenome editing can be translated to patients with imprinting disorders.

From oncogenesis to approval: the lentiviral gene therapy revolution in Wiskott-Aldrich syndrome.

This review traces the full arc of WAS gene therapy, from early preclinical proof of concept through regulatory approval, which culminated in the FDA approval of WASKYRA (etuvetidigene autotemcel) in December 2025, establishing autologous gene therapy as a licensed standard of care for WAS patients lacking a suitable H...

Long Non-Coding RNAs in Fish Reproduction: Emerging Regulators from Gonadal Fate to Fertility

Long non-coding RNAs (lncRNAs) have emerged as important regulators of gene expression acting through transcriptional, post-transcriptional, and epigenetic mechanisms. In teleosts, high-throughput sequencing has identified a growing number of lncRNAs associated with gonadal development and reproductive function, yet cu...

CRISPR/Cas9-Based Co-editing of KUP9 and CIPK9 Genes in Rapeseed (Brassica napus) to Improve Drought Tolerance and Yield Traits

Drought stress restricts rapeseed yield. KUP9 is involved in potassium ion transport and osmotic regulation whereas CIPK9 mediates abscisic acid signaling and stomatal movement which form an interaction network through potassium homeostasis and signal transduction. A dual-gene tandem expression cassette vector based on...

An integration-free CRISPR platform for genetic dissection of respiratory virus host dependencies in human nasal epithelium

Respiratory viruses, including coronaviruses, generally initiate infection in the upper respiratory tract, where mechanistic studies of host-virus interactions require models amenable to genetic manipulation. Human nasal air-liquid interface (ALI) cultures are realistic models of the nasal epithelium, but robust CRISPR...

Visual Narratives for Molecular Biology: Communicating Research, Methods, and Historical Experiments to Broad Audiences

INTRODUCTION Molecular biology investigates processes that occur at microscopic scales and are not directly observable, which often makes them difficult to communicate to non-specialist audiences. At the same time, advances such as gene editing, sequencing technologies, and molecular diagnostics are increasingly p...

Targeting the gene encoding human T-cell leukemia virus type 1 basic zip factor via CRISPR/Cas9 potentially affects ATL cell survival

Herein, we investigated the role of the human T-cell leukemia virus type 1 (HTLV-1)-encoded regulatory factor, HTLV-1 basic zip factor (HBZ), in HTLV-1 infections and adult T-cell leukemia/lymphoma (ATL). We designed five synthetic guide RNAs (sgRNAs) targeting HBZ and introduced them into ATL cell lines in vitro using...

How to build a mouse: embryo development captured in stunning detail

Targeting HPV16 E5 and E6 through gene-silencing approaches: mechanisms, preclinical evidence, and therapeutic perspectives

Cervical cancer, a significant global health challenge, is strongly associated with persistent infection with high-risk human papillomavirus (HPV), particularly HPV-16. The viral oncoproteins E5 and E6 are critical to HPV-mediated carcinogenesis, as they promote cell proliferation, immune evasion, apoptosis resistance,...

A universal twist-slide coupling mechanism governs deformations of DNA, RNA, and RNA-DNA hybrid duplexes

Nucleic acid duplexes, including DNA, RNA, and RNA–DNA hybrid (RDH), undergo structural deformations that are essential for genome replication, transcription, and RNA-mediated regulation. However, quantitative measurements and mechanistic understanding of these deformations remain limited. Here, we probe nucleic acid d...

Alzheimer's disease target and drug discovery by leveraging multiomics and electronic health data.

Despite prolific genomic data from individuals with Alzheimer's disease (AD), translation into treatments has lagged. Here we applied Mendelian randomization to multiple AD genome-wide association studies and functional multiomics datasets to nominate druggable targets, identifying 19 targets for individuals of Europea...