AAV Vectors in Molecular Therapy

Molecular therapy is the introduction of genetic material into cells to convey a biological benefit. Genetic material may be introduced to replace a missing or defective gene, or to add new genetic instructions to fight disease.

Types of molecular therapy include:

Gene replacement

• Replacement/addition of a normal gene, where a gene is defective or missing
• Typically used for inherited diseases
  • Example: Hemophilia B (deliver Factor IX gene)

Gene addition

• Expressing a therapeutic gene at the site needed
• Most often used in more complex disorders
  • Example: Peripheral arterial disease (deliver VEGF gene)

Modulation of gene expression: reducing effects of a deleterious gene product

• Silencing gene expression (RNAi/shRNA)
  • Example: Hepatitis B (deliver RNAi directed at Hepatitis B genes)