Controlled Muscle-Specific Gene Delivery

Treatment with the AAV gene therapy with doxycycline (B) induced expression of the target gene.

Invention Summary:

Adeno-associated virus (AAV) vector-based gene therapy has the potential to treat a number of rare diseases. However, only two AAV-based gene therapies have been approved by the FDA, primarily due to toxicity and lack of specificity.

Researchers at Rutgers have developed a novel method for designing AAV gene therapy with the ability to regulate gene expression in diseased tissues. It uses a muscle enhancer, muscle creatine kinase (MCK), and a specific promoter that controls the expression reverse by tetracycline transactivator (rtTA) in the presence of doxycycline. The therapy is only active when doxycycline is administered and can be modulated based on dose of doxycycline. Animal studies using the AAV technology demonstrated the expression can be controlled spatially and temporally in muscles with the presence of doxycycline.

Market Applications:

  • Gene therapy
  • Rare Diseases
  • Viral vector manufacturing


  • AAV Viral vector with high affinity towards muscle tissue.
  • Specific control of gene expression with the presence or absence of doxycycline.

Intellectual Property & Development Status: 

Patent pending. Available for licensing and/or research collaboration. Please contact

Patent Information:
Ryan Escolin
Licensing Manager, Life Sciences
Rutgers, The State University of New Jersey
Gene editing