rAAV-Regulatory Elements hGH polyA

The hGH polyadenylation (PolyA) signal is a regulatory element derived from the human growth hormone gene. It plays a crucial role in post-transcriptional regulation in gene therapy and recombinant adeno-associated virus (rAAV) systems. The hGH PolyA ensures efficient transcription termination, mRNA processing, and stable gene expression.

Structure and Role of hGH PolyA:

• Polyadenylation:
  • Adds a poly(A) tail to the 3' end of mRNA, protecting it from degradation.
• Transcription Termination:
  • Provides a termination signal for RNA polymerase II, ensuring clean transcript ends.
• Enhanced Expression:
  • Ensures proper splicing, export of mRNA to the cytoplasm, and efficient translation.
• Stability and Functionality:
  • Improves the stability of therapeutic mRNA and protein production.

Advantages of Using hGH PolyA in rAAV Vectors:

• High Efficiency:
  • Supports strong and reliable gene expression in various cell types and tissues.
• Human-Derived:
  • Reduces potential immunogenicity, making it particularly suitable for clinical applications.
• Broad Applicability:
  • Functional across multiple rAAV platforms, including tissue-specific and systemic gene delivery systems.

Applications in Gene Therapy:

• Therapeutic Protein Expression:
  • Used in rAAV vectors delivering proteins such as clotting factors (e.g., hemophilia treatments) or enzymes for metabolic disorders.
• Neurological Diseases:
  • Ensures stable transgene expression in neurons for conditions like Parkinson's disease and spinal muscular atrophy (SMA).
• Ophthalmic Gene Therapy:
  • Provides sustained expression for treating retinal degenerative diseases.

The hGH polyA signal is a cornerstone in rAAV vector design, offering robust and stable post-transcriptional regulation. Its effectiveness in supporting efficient and sustained gene expression makes it indispensable for advancing gene therapy technologies.