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New and emerging targeted therapies for cystic fibrosis.

Quon BS, Rowe SM.

BMJ. 2016 Mar 30;352:i859. doi: 10.1136/bmj.i859. Review.


Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

Alton EW, Armstrong DK, Ashby D, Bayfield KJ, Bilton D, Bloomfield EV, Boyd AC, Brand J, Buchan R, Calcedo R, Carvelli P, Chan M, Cheng SH, Collie DD, Cunningham S, Davidson HE, Davies G, Davies JC, Davies LA, Dewar MH, Doherty A, Donovan J, Dwyer NS, Elgmati HI, Featherstone RF, Gavino J, Gea-Sorli S, Geddes DM, Gibson JS, Gill DR, Greening AP, Griesenbach U, Hansell DM, Harman K, Higgins TE, Hodges SL, Hyde SC, Hyndman L, Innes JA, Jacob J, Jones N, Keogh BF, Limberis MP, Lloyd-Evans P, Maclean AW, Manvell MC, McCormick D, McGovern M, McLachlan G, Meng C, Montero MA, Milligan H, Moyce LJ, Murray GD, Nicholson AG, Osadolor T, Parra-Leiton J, Porteous DJ, Pringle IA, Punch EK, Pytel KM, Quittner AL, Rivellini G, Saunders CJ, Scheule RK, Sheard S, Simmonds NJ, Smith K, Smith SN, Soussi N, Soussi S, Spearing EJ, Stevenson BJ, Sumner-Jones SG, Turkkila M, Ureta RP, Waller MD, Wasowicz MY, Wilson JM, Wolstenholme-Hogg P; UK Cystic Fibrosis Gene Therapy Consortium.

Lancet Respir Med. 2015 Sep;3(9):684-91. doi: 10.1016/S2213-2600(15)00245-3. Epub 2015 Jul 3. Erratum in: Lancet Respir Med. 2015 Sep;3(9):e33.


Cystic Fibrosis Gene Therapy in the UK and Elsewhere.

Griesenbach U, Pytel KM, Alton EW.

Hum Gene Ther. 2015 May;26(5):266-75. doi: 10.1089/hum.2015.027. Review.


The fetal respiratory system as target for antenatal therapy.

Toelen J, Carlon M, Claus F, Gijsbers R, Sandaite I, Dierickx K, Devlieger R, Devriendt K, Debeer A, Proesmans M, Debyser Z, Deprest AJ.

Facts Views Vis Obgyn. 2011;3(1):22-35. Review.


The effects of lipoplex formulation variables on the protein corona and comparisons with in vitro transfection efficiency.

Betker JL, Gomez J, Anchordoquy TJ.

J Control Release. 2013 Nov 10;171(3):261-8. doi: 10.1016/j.jconrel.2013.07.024. Epub 2013 Aug 3.


Gene therapy for the treatment of cystic fibrosis.

Burney TJ, Davies JC.

Appl Clin Genet. 2012 May 29;5:29-36. doi: 10.2147/TACG.S8873. Print 2012.


Cholesterol domains enhance transfection.

Betker JL, Kullberg M, Gomez J, Anchordoquy TJ.

Ther Deliv. 2013 Apr;4(4):453-62. doi: 10.4155/tde.13.16.


Adenoviral gene transfer corrects the ion transport defect in the sinus epithelia of a porcine CF model.

Potash AE, Wallen TJ, Karp PH, Ernst S, Moninger TO, Gansemer ND, Stoltz DA, Zabner J, Chang EH.

Mol Ther. 2013 May;21(5):947-53. doi: 10.1038/mt.2013.49. Epub 2013 Mar 19.


Manganese-loaded lipid-micellar theranostics for simultaneous drug and gene delivery to lungs.

Howell M, Mallela J, Wang C, Ravi S, Dixit S, Garapati U, Mohapatra S.

J Control Release. 2013 Apr 28;167(2):210-8. doi: 10.1016/j.jconrel.2013.01.029. Epub 2013 Feb 6.


Ligands located within a cholesterol domain enhance gene delivery to the target tissue.

Xu L, Betker J, Yin H, Anchordoquy TJ.

J Control Release. 2012 May 30;160(1):57-63. doi: 10.1016/j.jconrel.2012.03.003. Epub 2012 Mar 9.


Gene therapy: light is finally in the tunnel.

Cao H, Molday RS, Hu J.

Protein Cell. 2011 Dec;2(12):973-89. doi: 10.1007/s13238-011-1126-y. Epub 2012 Jan 10. Review.


DNA as therapeutics; an update.

Saraswat P, Soni RR, Bhandari A, Nagori BP.

Indian J Pharm Sci. 2009 Sep;71(5):488-98. doi: 10.4103/0250-474X.58169.


Cellular endocytosis and gene delivery.

Ziello JE, Huang Y, Jovin IS.

Mol Med. 2010 May-Jun;16(5-6):222-9. doi: 10.2119/molmed.2009.00101. Epub 2010 Feb 3. Review.


The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways.

Griesenbach U, Meng C, Farley R, Wasowicz MY, Munkonge FM, Chan M, Stoneham C, Sumner-Jones SG, Pringle IA, Gill DR, Hyde SC, Stevenson B, Holder E, Ban H, Hasegawa M, Cheng SH, Scheule RK, Sinn PL, McCray PB Jr, Alton EW.

Biomaterials. 2010 Mar;31(9):2665-72. doi: 10.1016/j.biomaterials.2009.12.005. Epub 2009 Dec 21.


Detection of cystic fibrosis transmembrane conductance regulator activity in early-phase clinical trials.

Rowe SM, Accurso F, Clancy JP.

Proc Am Thorac Soc. 2007 Aug 1;4(4):387-98. Review.


Nonviral gene delivery: what we know and what is next.

Gao X, Kim KS, Liu D.

AAPS J. 2007 Mar 23;9(1):E92-104. Review.


Vectors for airway gene delivery.

Davis PB, Cooper MJ.

AAPS J. 2007 Jan 19;9(1):E11-7. Review.


Novel molecular approaches to cystic fibrosis gene therapy.

Lee TW, Matthews DA, Blair GE.

Biochem J. 2005 Apr 1;387(Pt 1):1-15. Review.


Preparation and property analysis of a hepatocyte targeting pH-sensitive liposome.

Wen SY, Wang XH, Lin L, Guan W, Wang SQ.

World J Gastroenterol. 2004 Jan 15;10(2):244-9.

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