Recent advancements in cancer therapy have led to the emergence of innovative approaches that offer new hope to patients. This review provides a comprehensive overview of the latest cutting-edge technologies and strategies in cancer treatment, including clinically approved therapies and experimental modalities in preclinical or early clinical development, with a focus on their potential to improve patient outcomes. We examined next-generation therapies, including personalized immunotherapies, targeted molecular treatments, gene editing approaches, and artificial intelligence-driven strategies. By evaluating the current landscape of these therapies, we highlighted their benefits, limitations, and future directions. The integration of these advanced modalities into clinical practice holds promise for enhancing their efficacy, reducing side effects, and ultimately transforming cancer care.

Neurofibromatosis type 1 (NF1) is the most common neurogenetic disorder worldwide, and its clinical presentations are highly variable. NF1 is caused by mutations in the NF1 gene, and 50% of NF1 cases are sporadic, which occur in the absence of a family history of the disease and usually result from a new mutation in the germline of a parent. Advanced paternal age may increase the risk for germinal NF1 mutations; however, some dominant conditions, including neurofibromatosis, have shown a lesser association with paternal age, although there are conflicting reports in the literature. We investigated the effects of paternal and maternal age in 241 NF1 patients (121 sporadic and 120 familial cases) who were seen in Hacettepe hospital, a reference center for genetic diseases in Turkey. For statistical analysis, Spearman’s and Chi-square tests were used. In this study, we evaluated paternal and maternal age at birth in sporadic and familial cases of NF1. We also compared the effect of parental age on the appearance and coexistence of various NF1 symptoms. There were no significant statistical differences between paternal age and coexistence of the NF1 symptoms. However, a slightly negative correlation was observed between paternal age and the coexistence of NF1 symptoms in familial cases (p < 0.05). We did not find strong evidence for the effect of parental age on the clinical severity of NF1.

Introduction: Nonketotic hyperglycinemia (NKH) is a rare, autosomal recessive-inherited disorder of amino acid metabolism known as glycine encephalopathy. Clinical manifestations arise because of the enzyme deficiency involved in glycine degradation. Currently, no effective treatment exists to alter the prognosis of NKH; available therapies focus primarily on reducing glycine accumulation in the body. MicroRNAs (miRNAs) are small noncoding RNAs that function as transcriptional and post-transcriptional regulators of gene expression. Here, we report the comparative profiling of small RNA sequencing (RNA-seq) data generated from clinical samples diagnosed with a specific condition. Methods: We identified miRNAs using miRNA-seq with samples obtained from three NKH patients, five individuals with heterozygous variants in NKH genes, and seven control cases. Utilising pathways from the PubChem database, we identified NKH-related pathways and used bioinformatics tools for miRNA, pathway, and disease prediction. This was followed by Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genomes (KEGG) analyses to enrich the predicted target genes of differentially expressed miRNAs based on miRNA-target interactions. Results: In our study, 10 known miRNAs were identified to be associated with NKH using at least two different tools. Our study is the first to demonstrate altered miRNA profiles in cases where the expression of AMT and GLDC genes is reduced. Conclusion: NKH is an ultrarare and difficult-to-diagnose disease. This study determines the miRNA-based biomarkers for early detection of NKH and provides a robust framework for advancing future experimental research and diagnostic strategies.

The expression of protein-coding genes is regulated by microRNAs (miRNAs), which are small non-coding RNAs typically 18-24 nucleotides in size. miRNAs have been found to play a critical role in regulating biological functions such as proliferation, differentiation, and development. MicroRNAs are transcribed by RNA polymerase II in the genome, and mature miRNAs bind to their target mRNA in the cytoplasm, thereby inhibiting translation. MicroRNAs are potential biomarkers for diseases and alternative therapeutic agents. A noninvasive test method with a low false-negative rate is required for the diagnosis of diseases in children. Along with discussing their potential use as biomarkers in pediatric diseases, this study aims to comprehensively examine miRNA research. The aim of this review is to emphasize that miRNAs can also serve as biomarkers and alternative therapeutic agents for pediatric diseases and to raise awareness.