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Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
Published on: March 16, 2022
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AAV gene therapy vectors in the TMJ.
Sabine M Brouxhon1, Michael Kerry O'Banion2, Stephanos Kyrkanides2,3
1Department of Physiology, School of Medicine, Stony Brook University, Stony Brook, New York, USA.
Clinical and Experimental Dental Research
|July 24, 2022
Summary
Gene therapy vectors adeno-associated viral vectors (AAV)-2 and AAV-6 show different tissue targeting in the temporomandibular joint (TMJ). This allows for precise gene delivery to specific cells for TMJ disorder treatments.
Area of Science:
- Gene Therapy
- Molecular Biology
- Orthopedics
Background:
- Temporomandibular joint (TMJ) disorders affect millions globally.
- Current treatments for TMJ disorders have limitations.
- Gene therapy offers a promising avenue for targeted TMJ disorder treatment.
Purpose of the Study:
- To evaluate adeno-associated viral vector (AAV)-2 and AAV-6 for gene delivery to the TMJ and its nerves.
- To determine the tissue tropism and infection efficacy of AAV-2 and AAV-6 in the TMJ.
- To assess the potential of these vectors for therapeutic gene delivery in TMJ disorders.
Main Methods:
- Intra-articular inoculation of wild-type C57BL/6 mice with AAV-2 and AAV-6 encoding the reporter gene GFP.
- Assessment of GFP expression in TMJ tissues (articular chondrocytes, meniscal fibroblasts) and trigeminal neurons.
- Characterization of viral vector tissue tropism and infection efficiency.
Main Results:
- AAV-2 and AAV-6 demonstrated distinct tissue tropism within the TMJ.
- AAV-2 infected articular chondrocytes, meniscal fibroblasts, and trigeminal neurons.
- AAV-6 exhibited selective tropism, primarily infecting neurons.
Conclusions:
- The study successfully evaluated AAV-2 and AAV-6 for gene delivery to the TMJ.
- Differential tropism of AAV-2 and AAV-6 allows for targeted gene delivery to specific TMJ tissues.
- This knowledge is clinically significant for developing targeted gene therapies for TMJ disorders.

