Misfolding & Disease

Protein Misfolding and Aggregation Kinetics

Summary

The kinetics of aggregation typically follow a sigmoidal curve characterized by a rate-limiting lag phase (primary nucleation), an exponential growth phase, and a plateau phase. Secondary nucleation on fibril surfaces is critical for toxic oligomer generation.

Key Points

  • 1Aggregation follows sigmoidal kinetics with lag, growth, and plateau phases
  • 2Secondary nucleation on fibrils generates toxic oligomers
  • 3Seeding eliminates the lag phase
  • 4Targeting secondary nucleation is key therapeutic strategy

Understanding the kinetics of protein aggregation is essential for developing therapeutic strategies against amyloid diseases.

The Sigmoidal Kinetic Profile

Amyloid formation characteristically follows a sigmoidal (S-shaped) curve:

Lag Phase

- Primary nucleation: Slow, thermodynamically unfavorable

  • Formation of initial nuclei is rate-limiting
  • Length varies dramatically between conditions
  • Can be eliminated by "seeding" with pre-formed fibrils
  • Growth Phase

  • Rapid, exponential increase in fibril mass
  • - Elongation: Monomers add to fibril ends

    - Secondary nucleation: New nuclei form on fibril surfaces

    Plateau Phase

  • Monomer concentration depleted
  • System reaches thermodynamic equilibrium
  • Fibrils continue to mature and reorganize
  • Microscopic Processes

    Modern kinetic analysis distinguishes multiple microscopic steps:

    Primary Nucleation

  • Spontaneous formation of nuclei from monomers
  • Typically requires 2-4 monomers
  • Very slow and unfavorable
  • Elongation

  • Addition of monomers to fibril ends
  • Relatively fast process
  • "Capping" fibril ends blocks elongation
  • Secondary Nucleation

    This is the critical insight of modern kinetic theory:

  • New nuclei form on the surface of existing fibrils
  • - Creates autocatalytic amplification

  • Produces the majority of toxic oligomers
  • Main target for therapeutic intervention
  • Fragmentation

  • Fibrils break to create new ends
  • Important in prion diseases
  • Mechanical agitation accelerates this
  • Therapeutic Implications

    Different kinetic stages offer different intervention points:

    1. Native state stabilizers: Prevent initial unfolding (e.g., Tafamidis)

    2. Primary nucleation inhibitors: Extend lag phase

    3. Secondary nucleation inhibitors: Reduce oligomer production

    4. Elongation inhibitors: Cap fibril ends

    5. Fibril breakers: Destabilize cross-β structure