In a phase 1a trial, one-time CRISPR-Cas9 gene editing with CTX310 was associated with no treatment-related dose-limiting toxic effects and with sustained reductions in ANGPTL3 and atherogenic lipids at 1 year.
Luke J. Laffin, S. Nicholls, Russell S Scott et al.· New England Journal of Medic...· 0 citations
Findings establish BrBGLU10 as an important regulator of pollen development and a potential target for fertility-related applications via gene editing in B. rapa and related Brassica crops.
Ying Huang, Shan-Xin Zhong, Tian-Ci Hu et al.· Plants· 0 citations
Whether that proposition that a pathogenic single-gene variant might be identified non-invasively at the earliest stage of pregnancy and corrected in situ before irreversible pathology develops is presently coherent as a therapeutic framework is examined.
S. Bittmann, E. Luchter, E. Moschüring-Alieva· Asian Journal of Medicine an...· 0 citations
Current therapeutic evidence is concentrated primarily in MMUT- deficient isolated MMA, and further progress will require safer and more durable delivery platforms, improved tissue targeting, robust long-term safety assessment, clinically meaningful endpoints, and careful evaluation of accessibility and cost.
Yan-Qiu Wang, Huifang Peng, T. Jiang et al.· Orphanet Journal of Rare Dis...· 0 citations
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This historical review presents the breakthrough discoveries by prominent Chinese geneticists during the early pioneering generation, the molecular genetics era, the transition to human and medical genetics, and current progress in genetic and genomic medicine.
Litao Bao, Yong-Hui Jiang, Yun-Fai Chris Lau et al.· Journal of Translational Gen...· 0 citations
A humanized multi-lineage lung model that captures epithelial-mesenchymal co-development and responsiveness to TGF-β1 stimulation and sensitivity to pirfenidone highlight its potential for antifibrotic drug screening.
Chen-Feng Hua, Chao-Jie Qu, Liu Yang et al.· Frontiers in Cell and Develo...· 0 citations
This study proposes a research and breeding roadmap progressing from molecular validation to medium- and short-term characterization in model trees and finally to multisite long-term ecological assessment, and presents a multiscale theoretical framework and testable pathways for translating SLs research from model plan...
Zhong-Zheng Ma, Wanxin Li, Qi Guo et al.· Physiologia Plantarum : An I...· 0 citations
This review comprehensively synthesizes recent advances in abiotic stress perception, signal transduction and hormone‐mediated regulation, highlighting their roles in shaping plant stress tolerance and proposes an integrated multi‐scale framework to provide a holistic understanding of drought and salinity stress tolera...
Muhammad Farooq, A. Khan, A. Hassan et al.· Plant Breeding· 1 citation
Recent advances in genetic and biomedical research under microgravity conditions are summarized, with particular emphasis on molecular mechanisms, omics technologies, genome editing, microbiome research, regenerative medicine, and personalized healthcare approaches.
Y. Hawsawi, Y. Jamous, Shouq F. Alghannam et al.· International Journal of Mol...· 0 citations
Overall, BRs represent promising targets for improving crop stress resilience; however, optimizing BR-mediated strategies and validating their long-term performance under diverse field conditions will be essential for their successful application in sustainable agriculture.
R. Jan, Shahzad Iqbal, Sajad Ali et al.· Plants· 1 citation
Overall, this review synthesizes the molecular basis of all four major EB types across 16+ classical genes, highlights the paradigm shift where NGS achieves a diagnostic yield exceeding 90%, and critically assesses recent therapeutic milestones—ranging from the first FDA-approved topical gene therapy to precision RNA a...
Maria Tsvetkova, T. Dimitrova, G. Yaneva· International Journal of Mol...· 0 citations
This review provides a comprehensive synthesis of a recent advances in CRISPR–Cas technologies and their strategic applications in crop genetics and hybrid breeding, and showcases how these technologies accelerate hybrid breeding by engineering male sterility systems, fixing heterosis, and generating high-throughput mu...
Syed Riaz Ahmed, Jahangir Khan, I. Ijaz et al.· Frontiers in Plant Science· 0 citations
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