Self-reactive CFTR T cells in humans: implications for gene therapy.

Journal article

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, nons...

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Authors: Calcedo, R; Griesenbach, U; Dorgan, D; Soussi, S; Boyd, A

• Publication status: Published
• Journal: Human gene therapy. Clinical development
• Volume: 24
• Issue: 3
• Pages: 108-115
• Publication date: 2013-09-01
• DOI: 10.1089/humc.2012.249
• EISSN: 2324-8645
• ISSN: 2324-8637
• Language: English
• Keywords: Humans; Interferon-gamma; Enzyme-Linked Immunosorbent Assay; Cystic Fibrosis Transmembrane Conductance Regulator; Middle Aged; Female; Interferon-gamma Release Tests; Male; Adolescent; Mice; Cystic Fibrosis; T-Lymphocytes; Genetic Therapy; Animals; Adult; Autoimmunity