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by Alton, Eric W F W, Prof and Armstrong, David K, MB ChB and Ashby, Deborah, Prof and Bayfield, Katie J, BSc and Bilton, Diana, Prof and Bloomfield, Emily V, BSc and Boyd, A Christopher, PhD and Brand, June, BSc and Buchan, Ruaridh, MPharm and Calcedo, Roberto, PhD and Carvelli, Paula, BN and Chan, Mario, MSc and Cheng, Seng H, PhD and Collie, D David S, MRCVS and Cunningham, Steve, MB ChB and Davidson, Heather E and Davies, Gwyneth, MBChB and Davies, Jane C, Prof and Davies, Lee A, DPhil and Dewar, Maria H, BSc and Doherty, Ann, BSc and Donovan, Jackie, PhD and Dwyer, Natalie S, RN and Elgmati, Hala I, MRCP and Featherstone, Rosanna F, BSc and Gavino, Jemyr, RN and Gea-Sorli, Sabrina, PhD and Geddes, Duncan M, Prof and Gibson, James S R, BSc and Gill, Deborah R, PhD and Greening, Andrew P, Prof and Griesenbach, Uta, Prof and Hansell, David M, Prof and Harman, Katharine, MRCPH and Higgins, Tracy E, BSc and Hodges, Samantha L, BSc and Hyde, Stephen C, DPhil and Hyndman, Laura, MSc and Innes, J Alastair, FRCPE and Jacob, Joseph, FRCR and Jones, Nancy, BPharm and Keogh, Brian F, FRCA and Limberis, Maria P, PhD and Lloyd-Evans, Paul, PhD and Maclean, Alan W, BSc and Manvell, Michelle C, BSc and McCormick, Dominique, PhD and McGovern, Michael, MRCP and McLachlan, Gerry, PhD and Meng, Cuixiang, BSc and Montero, M Angeles, MD and Milligan, Hazel, BSc and Moyce, Laura J, BSc and Murray, Gordon D, Prof and Nicholson, Andrew G, Prof and Osadolor, Tina, MSc and Parra-Leiton, Javier and Porteous, David J, Prof and Pringle, Ian A, DPhil and Punch, Emma K, BSc and Pytel, Kamila M, PhD and Quittner, Alexandra L, Prof and Rivellini, Gina and Saunders, Clare J, BSc and Scheule, Ronald K, PhD and Sheard, Sarah, FRCR and Simmonds, Nicholas J, FRCP and Smith, Keith, BSc and Smith, Stephen N, PhD and Soussi, Najwa, BSc and Soussi, Samia, BSc and Spearing, Emma J, MSc and Stevenson, Barbara J, BSc and Sumner-Jones, Stephanie G, PhD and Turkkila, Minna, BSc and Ureta, Rosa P, BN and Waller, Michael D, MRCP and Wasowicz, Marguerite Y, PhD and Wilson, James M, Prof and Wolstenholme-Hogg, Paul, PhD and UK Cystic Fibrosis Gene Therapy Co and UK Cystic Fibrosis Gene Therapy Consortium
Lancet Respiratory Medicine, The, ISSN 2213-2600, 2015, Volume 3, Issue 9, pp. 684 - 691
Journal Article
Molecular Therapy, ISSN 1525-0016, 11/2011, Volume 19, Issue 11, p. 1999
  In mice, liver-restricted expression of lysosomal enzymes from adeno-associated viral serotype 8 (AAV8) vectors results in reduced antibodies to the... 
Journal Article
Journal of Applied Physiology, ISSN 8750-7587, 05/2009, Volume 106, Issue 5, p. 1545
  Surfactant protein (SP)-D plays an important role in host defense and pulmonary surfactant homeostasis. In SP-D-deficient (Sftpd...) mice, the abnormal large... 
Proteins | Lungs | Rodents | Biochemistry | Surfactants | Cells | Binding sites
Journal Article
PLoS ONE, ISSN 1932-6203, 2010, Volume 5, Issue 11, p. e15033
Fabry disease is an X-linked glycosphingolipid storage disorder caused by a deficiency in the activity of the lysosomal hydrolase a-galactosidase A (a-gal).... 
MIGLUSTAT | SAFETY | INVOLVEMENT | BIOLOGY | REPLACEMENT THERAPY | RENAL-FUNCTION | MICE | AGALSIDASE-BETA THERAPY | GLOBOTRIAOSYLCERAMIDE | ALPHA-GALACTOSIDASE-A | GAUCHER-DISEASE | alpha-Galactosidase - genetics | alpha-Galactosidase - therapeutic use | Humans | Male | Chromatography, High Pressure Liquid | Mice, 129 Strain | Pyrrolidines - pharmacology | Pyrrolidines - therapeutic use | Mass Spectrometry | Uromodulin - urine | Female | Fabry Disease - drug therapy | Drug Therapy, Combination | Disease Models, Animal | Glucosyltransferases - antagonists & inhibitors | Trihexosylceramides - metabolism | Treatment Outcome | Fabry Disease - urine | Mice, Knockout | Drug Synergism | Enzyme Replacement Therapy - methods | Animals | Trihexosylceramides - urine | Fabry Disease - metabolism | Mice | Enzymes | Analysis | Physiological aspects | Kidney diseases | Drug therapy | Gangliosides | Biopharmaceutics | Heart | Globotriaosylceramide | Therapy | Syngeneic grafts | Liver | Galactosidase | Lysosomes | Biosynthesis | Tissues | Peripheral neuropathy | Accumulation | Ceramide glucosyltransferase | Reduction | Rodents | Heart diseases | Gaucher's disease | Recombinant | Urine | Pain perception | Effectiveness | Hydrolase | Medical treatment | Disease control | Coronary artery disease | Endothelial cells | Fabry's disease | Endothelium | Studies | Psychological aspects | Pathology | Glycosphingolipids | Storage | Biopsy | Product development
Journal Article
PLoS ONE, ISSN 1932-6203, 2011, Volume 6, Issue 6, p. e21758
Journal Article
Molecular Therapy, ISSN 1525-0016, 11/2010, Volume 18, Issue 11, pp. 1983 - 1994
Journal Article
PLoS ONE, ISSN 1932-6203, 08/2012, Volume 7, Issue 8, p. e43310
Journal Article
The Journal of Gene Medicine, ISSN 1099-498X, 01/2010, Volume 12, Issue 1, pp. 55 - 63
Journal Article
PLoS ONE, ISSN 1932-6203, 02/2013, Volume 8, Issue 2, p. e56181
Journal Article
Molecular Therapy, ISSN 1525-0016, 2006, Volume 13, Issue S1, pp. S269 - S269
  We have previously shown that high-frequency (1 MHz) ultrasound (U/S) increases naked plasmid DNA (pDNA) gene transfer in the mouse lung and that this effect... 
Journal Article