Aquaporin-1 gene transfer to correct radiation-induced salivary hypofunction

Handb Exp Pharmacol. 2009:(190):403-18. doi: 10.1007/978-3-540-79885-9_20.

Abstract

Irradiation damage to salivary glands is a common iatrogenic consequence of treatment for head and neck cancers. The subsequent lack of saliva production leads to many functional and quality-of-life problems for affected patients and there is no effective conventional therapy. To address this problem, we developed an in vivo gene therapy strategy involving viral vector-mediated transfer of the aquaporin-1 cDNA to irradiation-damaged glands and successfully tested it in two pre-clinical models (irradiated rats and miniature pigs), as well as demonstrated its safety in a large toxicology and biodistribution study. Thereafter, a clinical research protocol was developed that has received approval from all required authorities in the United States. Patients are currently being enrolled in this study.

Publication types

  • Research Support, N.I.H., Intramural
  • Review

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Aquaporin 1 / biosynthesis*
  • Aquaporin 1 / genetics
  • Cell Line
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Gene Transfer Techniques* / adverse effects
  • Genetic Therapy / adverse effects
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Humans
  • Radiation Injuries / etiology
  • Radiation Injuries / genetics
  • Radiation Injuries / metabolism
  • Radiation Injuries / therapy*
  • Radiotherapy / adverse effects
  • Research Design
  • Salivary Glands / metabolism*
  • Salivary Glands / radiation effects
  • Xerostomia / etiology
  • Xerostomia / genetics
  • Xerostomia / metabolism
  • Xerostomia / therapy*

Substances

  • AQP1 protein, human
  • Aquaporin 1