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female (4) 4
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leber congenital amaurosis - genetics (1) 1
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1. Full Text Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
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
Summary Background Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with... 
Pulmonary/Respiratory | DNA/LIPOSOMES | EFFICACY | SAFETY | RESPIRATORY SYSTEM | DNA | COMPLEXES | MUTATION | LUNGS | NASAL EPITHELIUM | AZITHROMYCIN | AEROSOL DELIVERY | CRITICAL CARE MEDICINE | Double-Blind Method | Forced Expiratory Volume - drug effects | Cystic Fibrosis - physiopathology | Humans | Administration, Inhalation | Male | United Kingdom | Young Adult | Cystic Fibrosis - genetics | Adolescent | Cystic Fibrosis Transmembrane Conductance Regulator - administration & dosage | Adult | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Female | Liposomes | Mutation | Child | Cystic Fibrosis - drug therapy | Nebulizers and Vaporizers | Plasmids - administration & dosage | Genetic Therapy - methods
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2. Full Text Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy
by Weleber, Richard G., MD and Pennesi, Mark E., MD, PhD and Wilson, David J., MD and Kaushal, Shalesh, MD, PhD and Erker, Laura R., PhD and Jensen, Lauren, MS, RN and McBride, Maureen T., BS and Flotte, Terence R., MD and Humphries, Margaret, RN and Calcedo, Roberto, PhD and Hauswirth, William W., PhD and Chulay, Jeffrey D., MD and Stout, J. Timothy, MD, PhD
Ophthalmology, ISSN 0161-6420, 2016, Volume 123, Issue 7, pp. 1606 - 1620
Purpose To provide an initial assessment of the safety of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in adults and children... 
Ophthalmology | CANINE MODEL | VISION | ELECTRORETINOGRAPHY | SAFETY | ROD | TRANSLUCENT | OPHTHALMOLOGY | RPE65 MUTATIONS | HUMAN CONE | PHOTORECEPTORS | DEGENERATION | Electroretinography | Dependovirus - genetics | Visual Fields - physiology | Leber Congenital Amaurosis - physiopathology | Humans | Male | Retinal Degeneration - etiology | Leber Congenital Amaurosis - genetics | Young Adult | cis-trans-Isomerases - genetics | Retinal Degeneration - therapy | Quality of Life | Adult | Female | Genetic Vectors | Child | Leber Congenital Amaurosis - therapy | Visual Acuity - physiology | Injections, Intraocular | Genetic Therapy - methods | Prevention | Care and treatment | Genetic disorders | Genetic vectors | Analysis | Blindness | Retinal degeneration | Gene therapy
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3. Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques
by Greig, Jenny A and Limberis, Maria P and Bell, Peter and Chen, Shu-Jen and Calcedo, Roberto and Rader, Daniel J and Wilson, James M
Human Gene Therapy Clinical Development, ISSN 2324-8637, 03/2017, Volume 28, Issue 1, pp. 39 - 50
Vectors based on adeno-associated virus serotype 8 (AAV8) have been evaluated in several clinical trials of gene therapy for hemophilia B with encouraging... 
toxicology | gene therapy | low density lipoprotein receptor | LDLR | AAV | familial hypercholesterolemia | MEDICINE, RESEARCH & EXPERIMENTAL | EFFICACY | SAFETY | MEDIATED GENE-TRANSFER | DEFICIENCY | RESPONSES | THERAPY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | HEMOPHILIA-B | LIVER | DENSITY-LIPOPROTEIN RECEPTOR | Dependovirus - genetics | Genetic Therapy | Hyperlipoproteinemia Type II - therapy | Receptors, LDL - genetics | Liver - pathology | Genetic Vectors - administration & dosage | Cholesterol - blood | Humans | Liver - metabolism | APOBEC-1 Deaminase - genetics | Male | Macaca mulatta | Mutation - genetics | APOBEC-1 Deaminase - deficiency | Animals | Receptors, LDL - deficiency | Genetic Vectors - toxicity | Female | Disease Models, Animal | Hyperlipoproteinemia Type II - genetics
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4. Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia
by Greig, Jenny A and Limberis, Maria P and Bell, Peter and Chen, Shu-Jen and Calcedo, Roberto and Rader, Daniel J and Wilson, James M
Human Gene Therapy Clinical Development, ISSN 2324-8637, 03/2017, Volume 28, Issue 1, pp. 28 - 38
The homozygous form of familial hypercholesterolemia (HoFH) is an excellent model for developing in vivo gene therapy in humans. The success of orthotropic... 
gene therapy | low density lipoprotein receptor | HoFH | LDLR | AAV | familial hypercholesterolemia | MEDICINE, RESEARCH & EXPERIMENTAL | ATHEROSCLEROSIS | PORTACAVAL-SHUNT | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | CHOLESTEROL LEVELS | GENE-THERAPY | MICE | VECTORS | DENSITY-LIPOPROTEIN RECEPTOR | Dependovirus - genetics | Genetic Therapy | Hyperlipoproteinemia Type II - therapy | Receptors, LDL - genetics | Liver - pathology | Genetic Vectors - administration & dosage | Cholesterol - blood | Humans | Liver - metabolism | Mice, Inbred C57BL | APOBEC-1 Deaminase - genetics | Male | Mice, Knockout | APOBEC-1 Deaminase - deficiency | Homozygote | Animals | Receptors, LDL - deficiency | Genetic Vectors - toxicity | Female | Mice | Disease Models, Animal | Hyperlipoproteinemia Type II - genetics
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