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Cystic Fibrosis Gene Therapy Research

Our Aims

• To contribute to the design of non-viral gene therapy and gene therapy trials in conjunction with our collaborators in the UK Cystic Fibrosis Gene Therapy Consortium
• To develop novel biochemical and clinical assays to assess gene transfer in patients with cystic fibrosis lung disease after gene therapy
• To identify novel biomarkers of infection and inflammation in cystic fibrosis lung disease

Projects

1. Detection of CFTR Following CF Gene Therapy
2. Serum Calprotectin as a Measure of Disease Activity in Cystic Fibrosis Lung Disease
3. Lung Clearance Index (LCI)
4. Development of novel messenger RNA (mRNA) assays for cystic fibrosis gene therapy
5. Paediatric
6. In vivo Gene Delivery
7. Investigation of microRNA expression in lung tissue
8. Novel Nonviral Transfection Reagents

Recent Publications

1. Folding and rescue of a cystic fibrosis transmembrane conductance regulator trafficking mutant identified using human-murine chimeric proteins.
   Da Paula AC, Sousa M, Xu Z, Dawson ES, Boyd AC, Sheppard DN, Amaral MD.
   J Biol Chem. 2010 Aug 27;285(35):27033-44. Epub 2010 Jun 15
2. Sputum and serum calprotectin are useful biomarkers during CF exacerbation.
   Gray RD, Imrie M, Boyd AC, Porteous D, Innes JA, Greening AP.
   J Cyst Fibros. 2010 May;9(3):193-8. Epub 2010 Mar 17.
3. Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.
   Holder E, Stevenson B, Farley R, Hilliard T, Wodehouse T, Somerton L, Larsen M, O'Donoghue J, Coles RL, Scheule RK, Cheng SH, Gill DR, Hyde SC, Griesenbach U, Alton EW, Porteous DJ, Boyd AC.
   J Gene Med. 2010 Jan;12(1):55-63.PMID: 19937989
4. SELDI-TOF biomarker signatures for cystic fibrosis, asthma and chronic obstructive pulmonary disease.
   Gomes-Alves P, Imrie M, Gray RD, Nogueira P, Ciordia S, Pacheco P, Azevedo P, Lopes C, de Almeida AB, Guardiano M, Porteous DJ, Albar JP, Boyd AC, Penque D.
   Clin Biochem. 2010 Jan;43(1-2):168-77. Epub 2009 Oct 20.PMID: 19850022
5. Sputum trace metals are biomarkers of inflammatory and suppurative lung disease.
   Gray RD, Duncan A, Noble D, Imrie M, O'Reilly DS, Innes JA, Porteous DJ, Greening AP, Boyd AC.
   Chest. 2010 Mar;137(3):635-41. Epub 2009 Oct 3.PMID: 19801580
6. Limitations of the murine nose in the development of nonviral airway gene transfer.
   Griesenbach U, Sumner-Jones SG, Holder E, Munkonge FM, Wodehouse T, Smith SN, Wasowicz MY, Pringle I, Casamayor I, Chan M, Coles R, Cornish N, Dewar A, Doherty A, Farley R, Green AM, Jones BL, Larsen MD, Lawton AE, Manvell M, Painter H, Singh C, Somerton L, Stevenson B, Varathalingam A, Siegel C, Scheule RK, Cheng SH, Davies JC, Porteous DJ, Gill DR, Boyd AC, Hyde SC, Alton EW.
   Am J Respir Cell Mol Biol. 2010 Jul;43(1):46-54. Epub 2009 Jul 31.PMID: 19648474