Abstract

This presentation focuses on gene therapy, an important and evolving tool in the new field of regenerative medicine. There are several clinical applications of gene therapy relevant for oral and maxillofacial surgery, one of which, the repair of radiation-damaged salivary glands, will be covered in depth. Initially, a general background in, and key considerations for, gene therapy will be provided, including different vectors that can be used and major characteristics of those most relevant. Secondly, there will be a general review of salivary gland biology and a brief description of radiation damage and its sequelae. Thirdly, the general strategy employed for repairing salivary gland radiation damage, transferring the aquaporin-1 gene (encoding a water channel protein) using an adenoviral vector, AdhAQP1, will be discussed, along with the presentation of detailed pre-clinical data to demonstrate the efficacy and general safety of the strategy. Next, a description of the development of a Phase 1 clinical trial using AdhAQP1 and initial findings will be given. Finally, there will be a discussion of problems that have arisen in gene therapy trials thus far and an assessment of its future clinical utility.

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