Cystic Fibrosis Gene Therapy Clinical Trials

Below are the main cystic fibrosis gene therapy clinical trials carried out by Consortium members.

1. Hyde SC, Southern KW, Gileadi U, Fitzjohn EM, Mofford KA, Waddell BE, Gooi HC, Goddard CA, Hannavy K, Smyth SE, Egan JJ, Sorgi FL, Huang L, Cuthbert AW, Evans MJ, Colledge WH, Higgins CF, Webb AK, Gill DR. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Ther 2000 Jul; 7(13): 1156-65.  View Abstract.

2. Alton EW, Stern M, Farley R, Jaffe A, Chadwick SL, Phillips J, Davies J, Smith SN, Browning J, Davies MG, Hodson ME, Durham SR, Li D, Jeffery PK, Scallan M, Balfour R, Eastman SJ, Cheng SH, Smith AE, Meeker D, Geddes DM. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet 1999 Mar 20; 353(9157): 947-54.  View Abstract.

3. Gill DR, Southern KW, Mofford KA, Seddon T, Huang L, Sorgi F, Thomson A, MacVinish LJ, Ratcliff R, Bilton D, Lane DJ, Littlewood JM, Webb AK, Middleton PG, Colledge WH, Cuthbert AW, Evans MJ, Higgins CF, Hyde SC. A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther 1997 Mar; 4(3): 199-209.  View Abstract.

4. Porteous DJ, Dorin JR, McLachlan G, Davidson-Smith H, Davidson H, Stevenson BJ, Carothers AD, Wallace WA, Moralee S, Hoenes C, Kallmeyer G, Michaelis U, Naujoks K, Ho LP, Samways JM, Imrie M, Greening AP, Innes JA. Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther 1997 Mar; 4(3): 210-18.  View Abstract.

5. Caplen NJ, Alton EW, Middleton PG, Dorin JR, Stevenson BJ, Gao X, Durham SR, Jeffery PK, Hodson ME, Coutelle C, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995 Jan;1(1):39-46.  View Abstract.

The Consortium has also published many other papers.  You can view a list of these papers here.

Selected Cystic Fibrosis Gene Therapy Related Editorials and Reviews

1. Alton EW. United Kingdom Cystic Fibrosis Gene Therapy Consortium. Use of nonviral vectors for cystic fibrosis gene therapy. Proc Am Thorac Soc. 2004;1(4):296-301.

2. Gill DR, Davies LA, Pringle IA, Hyde SC. The development of gene therapy for diseases of the lung. Cell Mol Life Sci. 2004 Feb;61(3):355-68.

3. Boyd AC, Porteous DJ. Revisiting the mouse lung model for CF. Gene Ther. 2004 May;11(9):737-8.

4. Griesenbach U, Geddes DM, Alton EW. Gene therapy for cystic fibrosis: an example for lung gene therapy. Gene Ther. 2004 Oct;11 Suppl 1:S43-50.

5. Griesenbach U, Geddes DM, Alton EW. Advances in cystic fibrosis gene therapy. Curr Opin Pulm Med. 2004 Nov;10(6):542-6.

6. Griesenbach U, Boyd AC; UK Cystic Fibrosis Gene Therapy Consortium. Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy. J Cyst Fibrosis. 2005 May;4(2):89-100.