Targeting disorders in unstructured and structured proteins in various diseases

Area of Science:

• Biochemistry
• Molecular Biology
• Drug Discovery

Background:

• Intrinsically disordered proteins (IDPs) and regions (IDPRs) lack stable structures, challenging traditional protein function theories.
• Their conformational flexibility allows specific yet low-affinity binding to multiple partners, crucial for signaling, recognition, and regulation.
• Dysregulation of IDPs/IDPRs is linked to various human diseases, including cancer, Parkinson's, and Alzheimer's disease.

Purpose of the Study:

• To review diseases associated with protein disorder.
• To summarize therapeutic targeting strategies for intrinsically disordered proteins and regions.
• To highlight the potential of targeting IDPs/IDPRs for novel drug design and discovery.

Main Methods:

• Literature review of protein disorder diseases.
• Analysis of therapeutic targeting methods for IDPs/IDPRs.
• Synthesis of current research on drug discovery for disordered proteins.

Main Results:

• IDPs/IDPRs are implicated in a wide range of human pathologies.
• Targeting IDPs/IDPRs presents unique challenges due to their dynamic nature.
• Various strategies are being developed for therapeutic intervention of disordered proteins.

Conclusions:

• Intrinsically disordered proteins and regions are critical players in human health and disease.
• Targeting IDPs/IDPRs represents a promising frontier for developing novel therapeutics.
• Successful targeting of these proteins could revolutionize rational drug design and discovery.