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Cystic fibrosis (CF) is one of the most common autosomal recessive lethal disorders affecting white populations of northern European ancestry. To date there is no cure for CF. Life-long treatments for CF are being developed and include gene therapy and the use of small-molecule drugs designed to target specific cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. Irrespective of the type of molecular therapy for CF, which may include gene replacement, exon skipping, nonsense suppression, or molecular correctors, because all of these modulate gene expression there is an inherent risk of activation of T cells against the wild-type version of CFTR. Here we report the validation of the human interferon-γ enzyme-linked immunospot assay and its application for the analysis of CFTR-specific T cell responses in patients with CF and in non-CF subjects. We found non-CF subjects with low levels of self-reactive CFTR-specific T cells in the United States and several patients with CF with low to high levels of self-reactive CFTR-specific T cells in both the United States and the United Kingdom.

Original publication

DOI

10.1089/humc.2012.249

Type

Journal article

Journal

Hum Gene Ther Clin Dev

Publication Date

09/2013

Volume

24

Pages

108 - 115

Keywords

Adolescent, Adult, Animals, Autoimmunity, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Enzyme-Linked Immunosorbent Assay, Female, Genetic Therapy, Humans, Interferon-gamma, Interferon-gamma Release Tests, Male, Mice, Middle Aged, T-Lymphocytes