Zobrazeno 1 - 8
of 8
pro vyhledávání: '"Kleine-Holthaus SM"'
Autor:
Liu W; UCL School of Pharmacy, University College London, UK., Kleine-Holthaus SM; UCL Institute of Ophthalmology, University College London, UK., Herranz-Martin S; UCL School of Pharmacy, University College London, UK; Centro de Biología Molecular Severo Ochoa (UAM-CSIC) and Departamento de Biología Molecular,Universidad Autónoma de Madrid (UAM), Madrid, Spain., Aristorena M; UCL Institute of Ophthalmology, University College London, UK., Mole SE; MRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK; UCL Great Ormond Street Institute of Child Health, 30 Guildford Street, London WC1N 1EH, UK., Smith AJ; UCL Institute of Ophthalmology, University College London, UK., Ali RR; UCL Institute of Ophthalmology, University College London, UK; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust, UK., Rahim AA; UCL School of Pharmacy, University College London, UK. Electronic address: a.rahim@ucl.ac.uk.
Publikováno v:
Biochimica et biophysica acta. Molecular basis of disease [Biochim Biophys Acta Mol Basis Dis] 2020 Sep 01; Vol. 1866 (9), pp. 165772. Date of Electronic Publication: 2020 Mar 24.
Autor:
Kleine Holthaus SM; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Aristorena M; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Maswood R; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Semenyuk O; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Hoke J; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Hare A; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Smith AJ; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom., Mole SE; MRC Laboratory for Molecular Cell Biology, University College London, London, United Kingdom.; UCL Institute of Child Health, London, United Kingdom.; UCL Department of Genetics, Evolution and Environment, University College London, London, United Kingdom., Ali RR; Department of Genetics, UCL Institute of Ophthalmology, London, United Kingdom.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2020 Jul; Vol. 31 (13-14), pp. 709-718.
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Autor:
Kleine Holthaus SM; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Herranz-Martin S; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK.; Centro de Biología Molecular Severo Ochoa (UAM-CSIC) and Departamento de Biología Molecular, Universidad Autónoma de Madrid (UAM), Madrid, Spain., Massaro G; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK., Aristorena M; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Hoke J; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Hughes MP; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK., Maswood R; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Semenyuk O; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Basche M; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Shah AZ; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Klaska IP; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Smith AJ; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Mole SE; MRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK.; UCL Institute of Child Health, 30 Guildford Street, London WC1N 1EH, UK.; UCL Department of Genetics, Evolution and Environment, University College London, Gower Street, London WC1E 6BT, UK., Rahim AA; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK., Ali RR; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust, London, UK.
Publikováno v:
Human molecular genetics [Hum Mol Genet] 2019 Dec 01; Vol. 28 (23), pp. 3867-3879.
Autor:
Mole SE; Medical Research Council Laboratory for Molecular Cell Biology and UCL Great Ormond Street Institute of Child Health, University College London, London, UK. Electronic address: s.mole@ucl.ac.uk., Anderson G; Department of Histopathology, Great Ormond Street Hospital, London, UK., Band HA; Batten Disease Family Association, Farnborough, UK., Berkovic SF; Epilepsy Research Centre, Department of Medicine, Austin Health & Northern Health, University of Melbourne, Melbourne, VIC, Australia., Cooper JD; Department of Pediatrics, Washington University School of Medicine, St Louis, MO, USA., Kleine Holthaus SM; UCL Institute of Ophthalmology, University College London, London, UK., McKay TR; Centre for Bioscience, Manchester Metropolitan University, Manchester, UK., Medina DL; Telethon Institute of Genetics and Medicine, Naples, Italy., Rahim AA; UCL School of Pharmacy, University College London, London, UK., Schulz A; Department of Pediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Smith AJ; UCL Institute of Ophthalmology, University College London, London, UK.
Publikováno v:
The Lancet. Neurology [Lancet Neurol] 2019 Jan; Vol. 18 (1), pp. 107-116. Date of Electronic Publication: 2018 Nov 21.
Autor:
Gonzalez-Cordero A; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Goh D; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Kruczek K; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Naeem A; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Fernando M; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Kleine Holthaus SM; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom .; 2 MRC Laboratory for Molecular Cell Biology, University College London, London, United Kingdom, United Kingdom ., Takaaki M; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Blackford SJI; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Kloc M; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Agundez L; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Sampson RD; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Borooah S; 3 Centre for Clinical Brain Sciences, University of Edinburgh , Edinburgh, United Kingdom ., Ovando-Roche P; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Mehat MS; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., West EL; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Smith AJ; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Pearson RA; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom ., Ali RR; 1 Department of Genetics, Institute of Ophthalmology, University College London, London, United Kingdom, United Kingdom .
Publikováno v:
Human gene therapy [Hum Gene Ther] 2018 Oct; Vol. 29 (10), pp. 1124-1139. Date of Electronic Publication: 2018 May 14.
Autor:
Kleine Holthaus SM; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; MRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK., Ribeiro J; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Abelleira-Hervas L; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Pearson RA; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Duran Y; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Georgiadis A; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Sampson RD; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Rizzi M; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Hoke J; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Maswood R; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Azam S; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Luhmann UFO; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Smith AJ; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Mole SE; MRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK; UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; UCL Department of Genetics, Evolution and Environment, University College London, Gower Street, London WC1E 6BT, UK. Electronic address: s.mole@ucl.ac.uk., Ali RR; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, City Road, London EC1V 2PD, UK. Electronic address: r.ali@ucl.ac.uk.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2018 May 02; Vol. 26 (5), pp. 1343-1353. Date of Electronic Publication: 2018 Mar 02.
Autor:
Kleine Holthaus SM; UCL Institute of Ophthalmology, Department of Genetics & NIHR BRC at Moorfields Eye Hospital, London, UK. s.kleine.holthaus.11@ucl.ac.uk.; MRC Laboratory for Molecular Cell Biology, UCL Institute of Child Health, Department of Genetics, Evolution and Environment, University College London, London, UK. s.kleine.holthaus.11@ucl.ac.uk., Smith AJ; UCL Institute of Ophthalmology, Department of Genetics & NIHR BRC at Moorfields Eye Hospital, London, UK., Mole SE; MRC Laboratory for Molecular Cell Biology, UCL Institute of Child Health, Department of Genetics, Evolution and Environment, University College London, London, UK., Ali RR; UCL Institute of Ophthalmology, Department of Genetics & NIHR BRC at Moorfields Eye Hospital, London, UK.
Publikováno v:
Advances in experimental medicine and biology [Adv Exp Med Biol] 2018; Vol. 1074, pp. 91-99.
Autor:
Chu CJ; School of Clinical Sciences, University of Bristol, Bristol BS8 1TD, UK colin.chu@bristol.ac.uk a.dick@bristol.ac.uk., Gardner PJ; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Copland DA; School of Clinical Sciences, University of Bristol, Bristol BS8 1TD, UK., Liyanage SE; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Gonzalez-Cordero A; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Kleine Holthaus SM; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Luhmann UF; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK Roche Pharmaceutical Research and Early Development, Ophthalmology Discovery & Biomarkers, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, Basel 4070, Switzerland., Smith AJ; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK., Ali RR; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK NIHR Biomedical Research Centre for Ophthalmology at Moorfields Eye Hospital and UCL Institute of Ophthalmology, London EC1V 9EL, UK., Dick AD; School of Clinical Sciences, University of Bristol, Bristol BS8 1TD, UK UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK NIHR Biomedical Research Centre for Ophthalmology at Moorfields Eye Hospital and UCL Institute of Ophthalmology, London EC1V 9EL, UK colin.chu@bristol.ac.uk a.dick@bristol.ac.uk.
Publikováno v:
Disease models & mechanisms [Dis Model Mech] 2016 Apr; Vol. 9 (4), pp. 473-81. Date of Electronic Publication: 2016 Jan 21.