Molecular Therapeutics

Lab Personnel

• Bing Wang, M.D., Ph.D., Lab Director
• Ying Tang
• M. Salay

Lab Projects

The Molecular Therapy Laboratory, under the direction of Bing Wang M.D., Ph.D., has focused on the virology of adeno-associated virus (AAV) and its utility as a promising vector system for gene therapy approaches targeting muscle, joint, bone, and cartilage. Recombinant AAV is endowed with distinctly advantageous biological properties including ease of integration into human chromosomes, nonpathogenicity, superior transduction of quiescent cells, long-term transgene expression, immuno-tolerance following gene transfer, non-toxicity after vector delivery, and acceptance by a wide range of hosts. Thus far, the AAV vector has demonstrated the most efficient gene transfer and longevity among all viral and non-viral vectors tested in the therapeutic application of rAAV for muscular dystrophies, muscle injury, bone defects, cartilage lesions, and rheumatoid arthritis.

In past years, Dr. Wang has been engaged in projects that concentrated on gene therapy for neuromuscular disorders, specifically Duchenne and Limb Girdle muscular dystrophies, using AAV viral vectors as gene vehicles. In addition to continued examination of Duchene muscular dystrophy , the long term plans for this lab involve the following research areas: chronic inflammatory arthritis, joint/tendon injuries, muscle/nerve injuries, skeletal muscle repair, and age-related bone loss and muscle atrophy. Currently, this lab is cooperating with other laboratories and making AAV constructs such as AAV-sFLT1, AAV-BMP4, AAV-Decorin, AAV-MPRO, AAV-MMP1, AAV-IKBSR, AAV- cFLIP ( University of Pittsburgh ), AAV- Osteoprotegerin ( Wayne State University ), and AAV-IKKα and -β (The Ohio State University).

Also, the lab is actively engaged in multiple applications of AAV vectors for gene therapy: i)Therapeutic gene packaging in self-complimentary AAV vectors; ii) siRNA in AAV vectors; iii) Tet on/Tet off AAV vector systems; iv)Purification and application of AAV vectors of different serotypes;and,v) S ystemic gene delivery of AAV into skeletal and cardiac muscles. Because the facility is equipped for AAV vector gene transfer studies and molecular biology, it is also a designated Vector Core for projects involving viral vector development for gene therapy. As a core laboratory, its goal is to construct adeno-associated-, adenoviral- and retroviral vectors for orthopaedic gene therapy research.

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