Abstract
The notion of gene therapy has been discussed for ~ 50 years, but it traditionally was considered suitable for use only with inherited monogenic disorders and cancers refractory to conventional therapy. More recently, the use of gene therapy (gene transfer) for other conditions, including quality of life disorders that lack a suitable treatment, has become accepted. This chapter describes the development of a gene therapy for one such quality of life disorder, the xerostomia caused by radiation-induced salivary gland hypofunction, from its initial idea to the conduct of a first in-human clinical trial.
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Acknowledgements
I have been extremely fortunate since the outset of the AdhAQP1 project to work with so many wonderful and talented colleagues and collaborators. I am sincerely appreciative to a great many people, but in particular I would like to thank Profs. Brian O’Connell, Christine Delporte and Songlin Wang for their critical contributions to the pre-clinical development of the AdhAQP1 strategy. Dr. Changyu Zheng and Ms. Corinne Goldsmith also played important roles in the pre-clinical studies, as well as were key members of the clinical study team. Drs. Ilias Alevizos, Ana Cotrim, Gabor Illei and Nikolai Nikolov, all core members of the clinical study team, were instrumental to the success of the clinical study, as were the two extremely talented Research Nurses for this study; Mss. Linda McCullagh and Shuying Liu. Additionally, I had the remarkable good fortune to work throughout most of my career in the Intramural Research Program of the National Institute of Dental and Craniofacial Research, which supported my studies from 1982 to 2011. Finally, I am especially indebted to the 11 research subjects, who participated in this first in human, salivary gland gene therapy study.
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Baum, B. (2015). Gene Therapy for Xerostomia. In: Sonis, DMD, DMSc, S. (eds) Genomics, Personalized Medicine and Oral Disease. Springer, Cham. https://doi.org/10.1007/978-3-319-17942-1_15
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DOI: https://doi.org/10.1007/978-3-319-17942-1_15
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