gene editing

Targeted insertion of an optimized donor DNA is effective in a humanized mouse model of dominant retinitis pigmentosa.

Retinitis pigmentosa (RP) affects 1 in 3,000 individuals worldwide, with 30%-40% of cases inherited as autosomal dominant (AD). Mutations in RHO (RP4) are the most common cause of ADRP. Because most RHO mutations exert gain-of-function or dominant-negative effects, conventional gene supplementation is insufficient, req...

Nanocarrier-enabled Gene and Nucleic Acid Therapeutics for Rheumatoid Arthritis: Advances, Translational Evidence, and Regulatory Challenges.

The available evidence underscores both the therapeutic potential and inherent complexity of nanocarrier-mediated gene and nucleic acid therapies for RA, emphasizing the necessity for optimized delivery strategies, rigorous safety assessments, and biomarker-guided patient stratification to achieve successful clinical t...

Integrative approaches to enhancing abiotic stress tolerance in wheat crop through physiological and molecular strategies

This review addresses the lack of an integrated synthesis connecting physiological traits, molecular mechanisms, and breeding strategies for multiple abiotic stresses in wheat and highlights emerging breeding technologies and climate-smart approaches to develop high-yielding, stress-resilient wheat cultivars that suppo...

Mitochondrial adenine base editing of mouse somatic tissues via adeno-associated viral delivery

The findings establish the feasibility of AAV-delivered mitochondrial A-to-G editing while defining important limitations that require further optimisation.

A CRISPR/Cas9 gene-editing platform for the rice leaffolder, Cnaphalocrocis medinalis.

A CRISPR/Cas9 toolkit was established for the rice leaffolder using entirely endogenous regulatory elements and provides a system for functional genomics in this pest.

Spacer-engineered donor DNA enhances CRISPR-Cas9-mediated knockin to establish a chemical knockdown platform for endogenous proteins.

A simple donor DNA engineering strategy that enhances HDR-mediated gene knockin by appending additional gRNA-recognizable spacer sequences to donor templates is investigated and established a readily implementable framework that integrates improved gene editing with on-demand chemical knockdown of endogenous proteins.

Novel and Emerging Therapies for Childhood-Onset Movement Disorders.

This work surveyed the literature, major conference proceedings, and expert networks to identify therapies approved, in clinical development, or supported by compelling preclinical data between 2022 and 2025, and focused on small molecules and genetic therapies for conditions in which movement disorders represent a pro...

Gene-editing therapies: delivering safely for patients.

Deciphering the Genetic Architecture of Whitefly (Bemisia tabaci) Resistance in Cotton: From Resistance Mechanisms to Molecular Breeding and Genome

One of the most significant natural fiber crops in the world is cotton (Gossypium hirsutum L.), but its productivity is severely limited by the whitefly Bemisia tabaci, a deadly phloem-feeding insect that triggers direct feeding damage and spreads Cotton Leaf Curl Disease (CLCuD), which results in significant yield and...

The hijacking of a wheat Exo70-like protein by a fungal pathogen triggers disease

It is demonstrated that elimination of the tan spot susceptibility gene Tsc2 through conventional breeding using a diagnostic marker or disruption of Tsc2 by gene editing can be used to improve tan spot resistance in wheat.

Viral Hepatitis: An Update on Biology, Pathogenesis and Diagnosis

Viral hepatitis remains one of the leading causes of liver-related morbidity and mortality worldwide, despite major advances in vaccination, antiviral therapy, and public health interventions. Hepatitis viruses A, B, C, D, and E differ in their genomic structure, modes of transmission, mechanisms of infection, and clin...

ZFIN/zfin: Release 1184

What's Changed ZFIN-10286: soften diff highlight + add gene-symbol columns to report by @rtaylorzfin in https://github.com/ZFIN/zfin/pull/1919 ZFIN-10373: Solr backup/restore: per-instance backup, restore from shared zfindb by @rtaylorzfin in https://github.com/ZFIN/zfin/pull/1929 ZFIN-10388: Fix manually named STRs ig...