Toward gene therapy for growth hormone deficiency via salivary gland expression of growth hormone.

ABSTRACT:

Objectives

Salivary glands are useful targets for gene therapeutics. After gene transfer into salivary glands, regulated secretory pathway proteins, such as human growth hormone, are secreted into saliva, whereas constitutive secretory pathway proteins, such as erythropoietin, are secreted into the bloodstream. Secretion of human growth hormone (hGH) into the saliva is not therapeutically useful. In this study, we attempted to redirect the secretion of transgenic hGH from the saliva to the serum by site-directed mutagenesis.

Materials and methods

We tested hGH mutants first in vitro with AtT20 cells, a model endocrine cell line that exhibits polarized secretion of regulated secretory pathway proteins. Selected mutants were further studied in vivo using adenoviral-mediated ge

SUBMITTER: Racz GZ 

PROVIDER: S-EPMC4050033 | biostudies-literature | 2015 Mar

REPOSITORIES: biostudies-literature