gene editing

Precision Medicine in Sickle Cell Disease: From Molecular Pathways to Curative Strategies.

Sickle cell disease (SCD) is a monogenic β-globin disorder characterized by hemoglobin S polymerization, hemolysis, and vaso-occlusion, yet it exhibits remarkable clinical heterogeneity. Despite advances in supportive care and disease-modifying therapies, substantial variability in severity, treatment response, and lon...

Loss of APOBEC1 disrupts RNA editing homeostasis and accelerates prion disease progression.

Chemically composed Cas9 (ChemComCas9) for spatio-temporal regulation and precision genome engineering applications.

Chemically composed Cas9 (ChemComCas9) has emerged as a highly versatile CRISPR enzyme, offering tunable, on-demand, and targeted control over genome engineering functions. Despite its programmable, sequence-specific activity, uncontrolled or prolonged exposure to Cas9 can lead to severe genotoxicity, restricting its e...

FECH negatively regulates protoporphyrin IX accumulation and shell coloration in the dwarf surf clam Mulinia lateralis

Shell color in mollusks, shaped by pigment synthesis and deposition, offers a readily observable natural trait. The dwarf surf clam Mulinia lateralis serves as an ideal bivalve model but has a uniform white shell, which limits its utility for genetic studies. In this study, we aimed to explore the feasibility of constr...

In-vivo CRISPR/Base-Editing “One-Shot” Therapies Targeting PCSK9

The background of PCSK9 as a target, the gene editing machinery and the preliminary results are described, and the unique safety challenges of this technology are explored and considerations for appropriate patient populations are proposed to maintain net positive benefits despite unknowns.

Reactivation of a loss-of-function SlGLK2 allele by frame-restoring genome editing in tomato

The potential of genome editing to restore the latent functions of pseudogenized genes in modern crops is demonstrated, offering a new approach for recovering traits lost during plant domestication or crop improvement.

ABE-mediated dual targeting of TOX and ARHGEF1 synergistically enhances antitumor activity of tumor-infiltrating lymphocytes in PDAC

Tumor-infiltrating lymphocytes (TILs) represent a promising immunotherapeutic modality in solid tumors, yet their clinical efficacy in pancreatic ductal adenocarcinoma (PDAC) remains severely limited by inefficient expansion and functional exhaustion. TILs were isolated from pancreatic cancer surgical specimens and wer...

Single-nucleotide genome editing uncovers the biological impact of rs16944 in the IL1B promoter

Interleukin-1β (IL-1β) exerts context-dependent effects in cancer immunity, promoting both inflammatory tumor progression and antitumor immune responses. The IL1B promoter single-nucleotide polymorphism rs16944 has been associated with poor prognosis in hepatocellular carcinoma, yet its functional impact on immune sign...

Effective-founder model for balancing clonal and sexual propagation in coral restoration: analysis code and supplemental workbook

This deposit is the computational component of An Effective-Founder Model for Balancing Clonal and Sexual Propagation in Coral Restoration (Winters, 2026, Evolutionary Applications, submitted). It reproduces every table, figure and numerical value in the manuscript. Coral restoration combines asexual fragmentation, whi...

Advancing the Translational Pipeline: From In Vivo and In Vitro 2D Models to Next-Generation Cell Culture Models for Amyotrophic Lateral Sclerosis Modelling and Drug Discovery

Background: Amyotrophic lateral sclerosis (ALS) is still a global health challenge, as there is no effective treatment due to the inadequacy of conventional experimental platforms for comprehending pathogenic mechanisms and developing therapy. Objectives: This review details the recent progress and clinical utility of...

CRISPR/Cas genome editing strategies to enhance abiotic stress tolerance in sugar crops

Sugar crops including sugarcane, sugar beets, and sweet sorghum, are fundamental for global sucrose supply and crucial feedstocks for bioethanol production and industrial bioproducts. However, yield and sucrose accumulation are significantly influenced by abiotic stresses, including drought, salinity/alkalinity, heat,...

Severity-dependent and sex-biased dysregulation of A-to-I RNA editing in COVID-19 reveals CTSS as a key regulatory target

SARS-CoV-2 infection causes diverse clinical outcomes, with men facing higher risks of severe disease. Adenosine-to-inosine (A-to-I) RNA editing mediated by ADAR enzymes governs innate antiviral immunity, yet its contribution to COVID-19 severity and sex-biased disease remains unclear. We performed epitranscriptomic pr...