Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy

Autor: Vink, CA, Counsell, JR, Perocheau, DP, Karda, R, Buckley, SMK, Brugman, MH, Galla, M, Schambach, A, McKay, Tristan, Waddington, SN, Howe, SJ

Lentiviral vector genomic RNA requires sequences that partially overlap wild-type HIV-1 gag and env genes for packaging into vector particles. These HIV-1 packaging sequences constitute 19.6% of the wild-type HIV-1 genome and contain functional cis e

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od_______128::e9592e0127cf5919d8f3600395b67355
https://e-space.mmu.ac.uk/618767/

Urokinase-type plasminogen activator (uPA) regulates invasion and matrix remodelling in colorectal cancer.

Autor: Micalet A; UCL Centre for 3D Models of Health and Disease, Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, Charles Bell House, 43-45 Foley Street, London W1W 7TS, United Kingdom.; Department of Mechanical Engineering, University College London, Gower Street, London WC1E 6BT, United Kingdom., Tappouni LJ; UCL Centre for Targeted Cancer Therapies, Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, Charles Bell House, 43-45 Foley Street, London W1W 7TS, United Kingdom., Peszko K; UCL Centre for 3D Models of Health and Disease, Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, Charles Bell House, 43-45 Foley Street, London W1W 7TS, United Kingdom., Karagianni D; Immune Regulation and Tumour Immunotherapy Group, UCL Cancer Institute, University College London, 72 Huntley Street, London WC1E 6DD, United Kingdom., Lam A; UCL Centre for 3D Models of Health and Disease, Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, Charles Bell House, 43-45 Foley Street, London W1W 7TS, United Kingdom., Counsell JR; UCL Centre for Targeted Cancer Therapies, Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, Charles Bell House, 43-45 Foley Street, London W1W 7TS, United Kingdom., Quezada SA; Immune Regulation and Tumour Immunotherapy Group, UCL Cancer Institute, University College London, 72 Huntley Street, London WC1E 6DD, United Kingdom., Moeendarbary E; Department of Mechanical Engineering, University College London, Gower Street, London WC1E 6BT, United Kingdom.; 199 Biotechnologies Ltd., Gloucester Road, London W2 6LD, United Kingdom., Cheema U; UCL Centre for 3D Models of Health and Disease, Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, Charles Bell House, 43-45 Foley Street, London W1W 7TS, United Kingdom.

Publikováno v: Matrix biology plus [Matrix Biol Plus] 2023 Nov 15; Vol. 19-20, pp. 100137. Date of Electronic Publication: 2023 Nov 15 (Print Publication: 2023).

Genomic investigations of unexplained acute hepatitis in children.

Autor: Morfopoulou S; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK.; Section for Paediatrics, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK., Buddle S; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Torres Montaguth OE; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Atkinson L; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Guerra-Assunção JA; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Moradi Marjaneh M; Section for Paediatrics, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK.; Section of Virology, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK., Zennezini Chiozzi R; University College London Mass Spectrometry Science Technology Platform, Division of Biosciences, University College London, London, UK., Storey N; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Campos L; Histopathology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Hutchinson JC; Histopathology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Counsell JR; Research Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, London, UK., Pollara G; Division of Infection and Immunity, University College London, London, UK., Roy S; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Venturini C; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Antinao Diaz JF; Research Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, London, UK., Siam A; Research Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, London, UK.; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, UK., Tappouni LJ; Research Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, London, UK., Asgarian Z; Research Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, London, UK., Ng J; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, UK., Hanlon KS; Research Department of Targeted Intervention, Division of Surgery and Interventional Science, University College London, London, UK., Lennon A; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., McArdle A; Section for Paediatrics, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK., Czap A; Division of Infection and Immunity, University College London, London, UK., Rosenheim J; Division of Infection and Immunity, University College London, London, UK., Andrade C; Histopathology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Anderson G; Histopathology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Lee JCD; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Williams R; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Williams CA; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Tutill H; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Bayzid N; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Martin Bernal LM; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Macpherson H; Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London, UK., Montgomery KA; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK.; Department of Neurodegenerative Disease, Queen Square Institute of Neurology, University College London, London, UK., Moore C; Wales Specialist Virology Centre, Public Health Wales Microbiology Cardiff, University Hospital of Wales, Cardiff, UK., Templeton K; Department of Medical Microbiology, Edinburgh Royal Infirmary, Edinburgh, UK., Neill C; Public Health Agency Northern Ireland, Belfast, UK., Holden M; School of Medicine, University of St. Andrews, St. Andrews, UK.; Public Health Scotland, Edinburgh, UK., Gunson R; West of Scotland Specialist Virology Centre, Glasgow, UK., Shepherd SJ; West of Scotland Specialist Virology Centre, Glasgow, UK., Shah P; Section for Paediatrics, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK., Cooray S; Section for Paediatrics, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK., Voice M; Micropathology Ltd, University of Warwick Science Park, Coventry, UK., Steele M; Micropathology Ltd, University of Warwick Science Park, Coventry, UK., Fink C; Micropathology Ltd, University of Warwick Science Park, Coventry, UK., Whittaker TE; Molecular and Cellular Immunology, Great Ormond Street Institute of Child Health, University College London, London, UK., Santilli G; Molecular and Cellular Immunology, Great Ormond Street Institute of Child Health, University College London, London, UK., Gissen P; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Kaufer BB; Institute of Virology, Freie Universität Berlin, Berlin, Germany., Reich J; Institute of Virology, Freie Universität Berlin, Berlin, Germany., Andreani J; Nuffield Department of Medicine, University of Oxford, Oxford, UK.; Centre Hospitalier Universitaire (CHU) Grenoble-Alpes, Grenoble, France., Simmonds P; Nuffield Department of Medicine, University of Oxford, Oxford, UK., Alrabiah DK; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK.; National Centre for Biotechnology, King Abdulaziz City for Science and Technology, Riyadh, Saudi Arabia., Castellano S; Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, UK.; University College London Genomics, University College London, London, UK., Chikowore P; Roslin Institute, University of Edinburgh, Edinburgh, UK., Odam M; Roslin Institute, University of Edinburgh, Edinburgh, UK., Rampling T; Division of Infection and Immunity, University College London, London, UK.; UK Health Security Agency, London, UK.; Hospital for Tropical Diseases, University College London Hospitals NHS Foundation Trust, London, UK., Houlihan C; Division of Infection and Immunity, University College London, London, UK.; UK Health Security Agency, London, UK.; Department of Clinical Virology, University College London Hospitals, London, UK., Hoschler K; UK Health Security Agency, London, UK., Talts T; UK Health Security Agency, London, UK., Celma C; UK Health Security Agency, London, UK., Gonzalez S; UK Health Security Agency, London, UK., Gallagher E; UK Health Security Agency, London, UK., Simmons R; UK Health Security Agency, London, UK., Watson C; UK Health Security Agency, London, UK., Mandal S; UK Health Security Agency, London, UK., Zambon M; UK Health Security Agency, London, UK., Chand M; UK Health Security Agency, London, UK., Hatcher J; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., De S; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Baillie K; Roslin Institute, University of Edinburgh, Edinburgh, UK., Semple MG; Pandemic Institute, University of Liverpool, Liverpool, UK.; Respiratory Medicine, Alder Hey Children's Hospital NHS Foundation Trust, Liverpool, UK., Martin J; Centre for Genomics and Child Health, The Blizard Institute, Queen Mary University of London, London, UK., Ushiro-Lumb I; NHS Blood and Transplant, Bristol, UK., Noursadeghi M; Division of Infection and Immunity, University College London, London, UK., Deheragoda M; King's College Hospital, London, UK., Hadzic N; King's College Hospital, London, UK., Grammatikopoulos T; King's College Hospital, London, UK., Brown R; Department of Cellular Pathology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK., Kelgeri C; Liver Unit, Birmingham Women's and Children's NHS Foundation Trust, Birmingham, UK., Thalassinos K; University College London Mass Spectrometry Science Technology Platform, Division of Biosciences, University College London, London, UK.; Institute of Structural and Molecular Biology, Division of Biosciences, University College London, London, UK.; Institute of Structural and Molecular Biology, Birkbeck College, University of London, London, UK., Waddington SN; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, UK.; Medical Research Council Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg, South Africa., Jacques TS; Histopathology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.; Developmental Biology and Cancer Department, Great Ormond Street Institute of Child Health, University College London, London, UK., Thomson E; Medical Research Council-University of Glasgow Centre for Virus Research, Glasgow, UK., Levin M; Section for Paediatrics, Department of Infectious Diseases, Faculty of Medicine, Imperial College London, London, UK., Brown JR; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Breuer J; Infection, Immunity and Inflammation Department, Great Ormond Street Institute of Child Health, University College London, London, UK. j.breuer@ucl.ac.uk.; Department of Microbiology, Virology and Infection Control, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. j.breuer@ucl.ac.uk.

Publikováno v: Nature [Nature] 2023 May; Vol. 617 (7961), pp. 564-573. Date of Electronic Publication: 2023 Mar 30.

Dystrophin involvement in peripheral circadian SRF signalling.

Autor: Betts CA; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK corinne.betts@paediatrics.ox.ac.uk., Jagannath A; Sleep and Circadian Neuroscience Institute (SCNi), Nuffield Department of Clinical Neurosciences, Oxford Molecular Pathology Institute, Dunn School of Pathology, University of Oxford, Oxford, UK., van Westering TL; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., Bowerman M; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK.; School of Medicine, Keele University, Staffordshire, Wolfson Centre for Inherited Neuromuscular Disease, The Robert Jones and Agnes Hunt Orthopaedic Hospital, Oswestry, UK., Banerjee S; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., Meng J; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, University College London Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Falzarano MS; Department of Medical Sciences, Unit of Medical Genetics, University of Ferrara, Ferrara, Italy., Cravo L; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., McClorey G; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., Meijboom KE; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., Bhomra A; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., Lim WF; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., Rinaldi C; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK.; Muscular Dystrophy UK Oxford Neuromuscular Centre, University of Oxford, Oxford, UK., Counsell JR; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, University College London Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Chwalenia K; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK., O'Donovan E; Medical Research Council, Laboratory of Molecular Biology, Francis Crick Avenue, Cambridge, UK., Saleh AF; Medical Research Council, Laboratory of Molecular Biology, Francis Crick Avenue, Cambridge, UK.; Functional and Mechanistic Safety, Clinical Pharmacology and Safety Sciences, R&D, AstraZeneca, Cambridge, UK., Gait MJ; Medical Research Council, Laboratory of Molecular Biology, Francis Crick Avenue, Cambridge, UK., Morgan JE; Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, University College London Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Ferlini A; Department of Medical Sciences, Unit of Medical Genetics, University of Ferrara, Ferrara, Italy., Foster RG; Sleep and Circadian Neuroscience Institute (SCNi), Nuffield Department of Clinical Neurosciences, Oxford Molecular Pathology Institute, Dunn School of Pathology, University of Oxford, Oxford, UK., Wood MJ; Department of Paediatrics, University of Oxford, South Parks Road, Oxford, UK.; Muscular Dystrophy UK Oxford Neuromuscular Centre, University of Oxford, Oxford, UK.

Publikováno v: Life science alliance [Life Sci Alliance] 2021 Aug 13; Vol. 4 (10). Date of Electronic Publication: 2021 Aug 13 (Print Publication: 2021).

Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.

Autor: Ng J; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK.; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK., Barral S; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK. s.waddington@ucl.ac.uk s.barral@ucl.ac.uk., De La Fuente Barrigon C; Genetics and Genomic Medicine, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK., Lignani G; Clinical and Experimental Epilepsy, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK., Erdem FA; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK.; Institute of Pharmacology and Gaston H. Glock Laboratories for Exploratory Drug Research, Centre of Physiology and Pharmacology, Medical University of Vienna, 1090 Vienna, Austria., Wallings R; Oxford Parkinson's Disease Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, OX1 3PT, UK., Privolizzi R; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK.; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK., Rossignoli G; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK., Alrashidi H; Genetics and Genomic Medicine, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK., Heasman S; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK., Meyer E; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK., Ngoh A; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK., Pope S; Neurometabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, WC1N 3BG, UK., Karda R; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK., Perocheau D; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK., Baruteau J; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK.; Genetics and Genomic Medicine, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK., Suff N; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK.; Department of Women and Children's Health, King's College London, London, WC2R 2LS, UK., Antinao Diaz J; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK., Schorge S; Clinical and Experimental Epilepsy, Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.; Pharmacology, School of Pharmacy, University College London, London, WC1N 1AX, UK., Vowles J; James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, OX1 3RE, UK., Marshall LR; Infection, Immunity, Inflammation, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK., Cowley SA; James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, OX1 3RE, UK., Sucic S; Institute of Pharmacology and Gaston H. Glock Laboratories for Exploratory Drug Research, Centre of Physiology and Pharmacology, Medical University of Vienna, 1090 Vienna, Austria., Freissmuth M; Institute of Pharmacology and Gaston H. Glock Laboratories for Exploratory Drug Research, Centre of Physiology and Pharmacology, Medical University of Vienna, 1090 Vienna, Austria., Counsell JR; Developmental Neurosciences, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK., Wade-Martins R; Oxford Parkinson's Disease Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, OX1 3PT, UK., Heales SJR; Genetics and Genomic Medicine, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK.; Neurometabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, WC1N 3BG, UK., Rahim AA; Pharmacology, School of Pharmacy, University College London, London, WC1N 1AX, UK., Bencze M; Developmental Neurosciences, GOS-Institute of Child Health, University College London, London, WC1N 1EH, UK.; University Paris Est Creteil, INSERM, IMRB, 94000 Creteil, France., Waddington SN; Gene Transfer Technology Group, EGA-Institute for Women's Health, University College London, London, WC1E 6HX, UK. s.waddington@ucl.ac.uk s.barral@ucl.ac.uk.; Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, 2193 Johannesburg, South Africa., Kurian MA; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, WC1N 1DZ, UK.; Department of Neurology, Great Ormond Street Hospital for Children, London, WC1N 3JH, UK.

Publikováno v: Science translational medicine [Sci Transl Med] 2021 May 19; Vol. 13 (594).