Journal of Gene Medicine最新文献

Ongoing developments in genome editing most notably the continued evolution of CRISPR-Cas systems and their orthogonal or modified counterparts have substantively altered both experimental and applied practices in biomedicine, agriculture, and therapeutic design. More recently, the systematic incorporation of artificial intelligence and machine learning methodologies has augmented the specificity, throughput, and explanatory capacity of genome-editing workflows, thereby refining the prediction of on-target efficiencies, the appraisal of off-target liabilities, and the tailoring of molecular therapeutic configurations. The present contribution offers an integrative survey of these computational developments, emphasizing (i) predictive algorithms, (ii) machine-learning and deep-learning frameworks, (iii) data-centric procedural strategies, and (iv) dedicated applications in oncology, neurology, rare-disease research, and precision-medicine contexts. Throughout, we evaluate architectural choices, sequence-encoding representations, and lingering dataset-related biases, while additionally addressing current constraints concerning model interpretability, ethical viability, and the procedural prerequisites for clinical translation. Moreover, we advance a structured taxonomy that organizes AI-mediated genome-editing approaches according to methodological lineage and functional scope, and we delineate extant research lacunae. By combining these elements, we supply a prospective assessment of the means by which artificial intelligence might be further leveraged to support secure, efficacious, and equitably accessible genome engineering outcomes.

Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histiocytosis identified in the early 20th century. ECD primarily affects adults and is characterized by systemic infiltration of foamy macrophages. ECD can infiltrate the bones, skin, heart, and central nervous system, causing various symptoms. Limited targeted treatments, immunotherapy, and chemotherapy options underscore the need for tailored approaches to this complex disorder. Recent studies have highlighted the role of microRNAs (miRNAs) as critical regulators of gene expression involved in various pathophysiological processes, including inflammation and immune responses. Likewise, ECD patients exhibited abnormal expression of miRNAs that can disrupt immunological checkpoints and histiocyte activity, leading to ECD histiocyte proliferation and survival. This review addresses the emerging evidence regarding the involvement of miRNAs in ECD, focusing on their potential as biomarkers for diagnosis and prognosis and their role in modulating the disease's inflammatory pathways. Understanding the specific miRNA profiles in ECD could provide insights into disease mechanisms and pave the way for targeted therapeutic strategies. Future research should elucidate the precise functions of miRNAs in ECD and their potential clinical applications.

Preeclampsia (PE) is a common complication during pregnancy. Trophoblast cells are the main cell type of the placenta, and abnormalities in proliferation and differentiation cause anterior placental accretion and lead to the development of PE. This study will investigate the function and related mechanism of N6-methyladenosine (m⁶A) reader YTH N6-methyladenosine RNA binding protein F3 (YTHDF3) in PE and provide a rationale for the therapeutic treatment of PE. In the study, YTHDF3 and suppressor of cytokine signaling 1 (SOCS1) expression were significantly upregulated within PE placental tissue samples. YTHDF3 knockdown promoted the capacity of hypoxia-treated trophoblast cells to proliferate, migrate, invade, and undergo epithelial-mesenchymal transition (EMT) and inhibited apoptosis. The JAK1/STAT3 pathway is aberrantly activated in trophoblast cells of PE. YTHDF3 binds m⁶A-modified SOCS1 mRNA to enhance its stability and translation. YTHDF3/SOCS1 promotes disease progression in PE by inhibiting the JAK1/STAT3 signaling pathway. In conclusion, YTHDF3 promotes the disease progression of PE by enhancing the m⁶A modification of SOCS1 mRNA and suppressing the JAK1/STAT3 signaling pathway. YTHDF3 has been identified as an underlying target for the clinical treatment of PE.