About this Research Topic
Hereditary diseases are a group of complex diseases caused by genetic variations, characterized by diverse etiologies, high genetic predisposition and family clustering. With the rapid advancement of genomics, molecular biology and biotechnology, human understanding of hereditary diseases has significantly deepened; however, treating these diseases remains a formidable challenge. It is therefore crucial to strengthen research into small molecules and protein targets for the treatment of genetic diseases. Small molecule drugs have long been at the forefront of drug research and development. In the treatment of genetic diseases, small molecule drugs can modulate cell signaling, metabolism, and various life processes by binding to specific proteins, demonstrating considerable application potential.
Small molecule and target research has become a major driver in the development of new medicines, the treatment of disease, and the improvement of human health. Research in this field not only unravels the mysteries of life science but also provides a solid theoretical foundation and technical support for drug design and development. Target proteins, being the direct focus of drug action, play a crucial role in disease initiation and progression. They are often key molecules in disease-related signaling pathways and influence disease progression by regulating biological processes such as cell proliferation, differentiation and apoptosis. Therefore, in-depth studies of the structure and function of target proteins is of great importance for elucidating disease mechanisms and discovering new therapeutic targets. Through high-throughput screening, structural biology, and computational simulation, scientists can identify target proteins that are closely associated with diseases and further investigate their interaction mechanisms with small molecule drugs.
Natural plants are the largest repository of biological resources on earth, rich in small molecule natural medicines. These small molecule compounds, with their diverse chemical structures and extensive biological activities, are an important source for drug discovery and development. By examining the interactions between small molecules and target proteins, the screening and characterization of these interactions can lead to the discovery of new drugs with potential medical value. Additionally, studying the mechanism of interaction between these small molecules and target proteins in detail can help identify new therapeutic targets, optimize drug design strategies, improve drug efficacy, and reduce toxic side effects.
We welcome submissions of original research papers, review, hypothesis & theory, methods, and opinion, including (but not limited to) research on the following sub-themes:
• Screening of active ingredients in traditional Chinese medicines for the treatment of related hereditary diseases
• Exploring the relationship between natural small molecules and the function of relevant target proteins in genetic diseases
• Investigating recent trends in small molecules and target proteins related to genetic diseases, including novel small molecules, targets, or pathways
• Conducting systematic evaluation or meta-analysis of the efficacy of specific small compounds against the above conditions and their underlying mechanisms.
Please note:
If patient data are analyzed, a comprehensive description of the patients including sex, age, diagnostic criteria, inclusion and exclusion criteria, disease stage, therapy received, comorbidities as well as additional clinical information and assessment of clinical response/effects should be included.
If genetic, proteomics, metabolomics, or other omics data are analyzed, a comprehensive description of the methods and the rationale for the selection of the specific data studied should be provided.
To include in this Research Topic studies on molecules obtained from natural compounds, herbal extracts, or traditional medicines, these molecules should be highly purified, have a known chemical structure, and tested isolated and at known concentrations to ensure reproducibility.
Studies solely based on the analysis of public databases or published evidence, with no further experimental insights or experimental validation, will not be included in this Research Topic.
Small molecule and target research has become a major driver in the development of new medicines, the treatment of disease, and the improvement of human health. Research in this field not only unravels the mysteries of life science but also provides a solid theoretical foundation and technical support for drug design and development. Target proteins, being the direct focus of drug action, play a crucial role in disease initiation and progression. They are often key molecules in disease-related signaling pathways and influence disease progression by regulating biological processes such as cell proliferation, differentiation and apoptosis. Therefore, in-depth studies of the structure and function of target proteins is of great importance for elucidating disease mechanisms and discovering new therapeutic targets. Through high-throughput screening, structural biology, and computational simulation, scientists can identify target proteins that are closely associated with diseases and further investigate their interaction mechanisms with small molecule drugs.
Natural plants are the largest repository of biological resources on earth, rich in small molecule natural medicines. These small molecule compounds, with their diverse chemical structures and extensive biological activities, are an important source for drug discovery and development. By examining the interactions between small molecules and target proteins, the screening and characterization of these interactions can lead to the discovery of new drugs with potential medical value. Additionally, studying the mechanism of interaction between these small molecules and target proteins in detail can help identify new therapeutic targets, optimize drug design strategies, improve drug efficacy, and reduce toxic side effects.
We welcome submissions of original research papers, review, hypothesis & theory, methods, and opinion, including (but not limited to) research on the following sub-themes:
• Screening of active ingredients in traditional Chinese medicines for the treatment of related hereditary diseases
• Exploring the relationship between natural small molecules and the function of relevant target proteins in genetic diseases
• Investigating recent trends in small molecules and target proteins related to genetic diseases, including novel small molecules, targets, or pathways
• Conducting systematic evaluation or meta-analysis of the efficacy of specific small compounds against the above conditions and their underlying mechanisms.
Please note:
If patient data are analyzed, a comprehensive description of the patients including sex, age, diagnostic criteria, inclusion and exclusion criteria, disease stage, therapy received, comorbidities as well as additional clinical information and assessment of clinical response/effects should be included.
If genetic, proteomics, metabolomics, or other omics data are analyzed, a comprehensive description of the methods and the rationale for the selection of the specific data studied should be provided.
To include in this Research Topic studies on molecules obtained from natural compounds, herbal extracts, or traditional medicines, these molecules should be highly purified, have a known chemical structure, and tested isolated and at known concentrations to ensure reproducibility.
Studies solely based on the analysis of public databases or published evidence, with no further experimental insights or experimental validation, will not be included in this Research Topic.
Keywords: Molecular docking, Dynamic simulation, Mechanism of action, Gene, Protein, molecular signaling, bioactive compounds
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
