International Journal of Molecular Medical Science, 2025, Vol.15, No.1, 20-32 http://medscipublisher.com/index.php/ijmms 28 over the lifetime of CF patients without causing additional health issues (Kramer and Clancy, 2018; Bakaj and Pocai, 2023). 8 Future Perspectives in CF Research 8.1 Integration of personalized medicine approaches, including genetic and metabolic profiling The integration of personalized medicine in Cystic Fibrosis (CF) research is poised to revolutionize patient care by tailoring treatments based on individual genetic and metabolic profiles. Genetic studies have already highlighted the complex genotype-phenotype relationships in CF, revealing that not all CFTR mutations result in the same clinical manifestations. This understanding has paved the way for personalized treatments that target specific genetic mutations, significantly improving patient outcomes (O’Neal and Knowles, 2018). Additionally, the advent of omics technologies, such as metabolomics and proteomics, has enabled the detailed profiling of metabolic pathways in CF patients. These approaches have identified distinct metabolic signatures associated with disease severity and progression, offering new avenues for targeted therapeutic interventions (Eiserich et al., 2012; Muhlebach and Sha, 2015). Metabolic profiling, in particular, has shown promise in identifying biomarkers that can predict disease progression and response to therapy. For instance, studies have demonstrated that lipid metabolism is significantly altered in CF, with specific lipid profiles correlating with lung function and infection status (Eiserich et al., 2012; Muhlebach and Sha, 2015). By integrating genetic and metabolic data, researchers can develop more precise and effective treatment strategies, ultimately improving the quality of life for CF patients. 8.2 Use of advanced models to study CF metabolism Advanced models, such as organoids and animal models, are invaluable tools for studying the metabolic pathways involved in CF and testing potential therapeutic interventions. Organoids, which are three-dimensional cultures derived from stem cells, closely mimic the structure and function of human tissues. These models have been used to study CFTR function and screen for drugs that can correct CFTR defects, providing a more accurate representation of human disease compared to traditional cell cultures (Rosen et al., 2017). Additionally, the use of CRISPR/Cas9 genome editing has enabled the creation of more precise CF models, allowing for the study of specific genetic mutations and their impact on disease pathology (Rosen et al., 2017). Animal models, including both genetic and non-genetic models, have also been instrumental in advancing our understanding of CF. These models help elucidate the mechanisms of disease pathogenesis and evaluate the efficacy of new treatments. For example, non-genetic models, such as infection-based and pharmacological models, can replicate specific aspects of CF pathology, providing insights into the complex interactions between host and pathogen (Leenaars et al., 2021). The continued development and refinement of these advanced models will be crucial for uncovering new therapeutic targets and improving existing treatments for CF. 8.3 Potential role of microbiome modulation in metabolic and immune regulation The role of the microbiome in CF is an emerging area of research that holds significant potential for therapeutic intervention. The CF respiratory tract is characterized by chronic infections and inflammation, which are influenced by the composition and activity of the microbial community (Eiserich et al., 2012). Modulating the microbiome to promote beneficial bacteria and reduce pathogenic species could help regulate metabolic and immune responses, potentially alleviating some of the disease's symptoms. Recent studies have shown that the microbiome can impact various metabolic pathways in CF, including those related to lipid metabolism and inflammation (Eiserich et al., 2012; Muhlebach and Sha, 2015). For instance, specific bacterial metabolites have been associated with clinical parameters such as lung function and infection status, suggesting that targeting these microbial pathways could have therapeutic benefits (Muhlebach and Sha, 2015). Additionally, the use of probiotics and other microbiome-modulating therapies is being explored as a means to enhance immune regulation and reduce inflammation in CF patients. Further research into the complex interactions between the microbiome, metabolism, and immune system will be essential for developing effective microbiome-based therapies for CF.
RkJQdWJsaXNoZXIy MjQ4ODYzNA==