Browse by UCL people
Group by: Type | Date
Number of items: 75.
2025
Geard, AF;
Massaro, G;
Hughes, MP;
Arbuthnot, P;
Waddington, SN;
Rahim, AA;
(2025)
Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy.
Pharmaceutics
, 17
(5)
, Article 650. 10.3390/pharmaceutics17050650.
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Sartorelli, J;
Ng, J;
Rahim, AA;
Waddington, SN;
Kurian, MA;
(2025)
Genetic therapies for movement disorders - current status.
Journal of Neurology
, 272
, Article 220. 10.1007/s00415-025-12940-5.
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2024
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Chu, Wing Sum;
Ng, Joanne;
Waddington, Simon N;
Kurian, Manju A;
(2024)
Gene therapy for neurotransmitter-related disorders.
Journal of Inherited Metabolic Diseases
, 47
(1)
pp. 176-191.
10.1002/jimd.12697.
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Massaro, Giulia;
Geard, Amy F;
Nelvagal, Hemanth R;
Gore, Katrina;
Clemo, Nadine K;
Waddington, Simon N;
Rahim, Ahad A;
(2024)
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
Human Molecular Genetics
, Article ddae081. 10.1093/hmg/ddae081.
(In press).
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Touramanidou, Loukia;
Gurung, Sonam;
Cozmescu, Claudiu A;
Perocheau, Dany;
Moulding, Dale;
Finn, Patrick F;
Frassetto, Andrea;
... Baruteau, Julien; + view all
(2024)
Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice.
Cells
, 13
(23)
, Article 1979. 10.3390/cells13231979.
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Wetzel, Andrea;
Lei, Si Hang;
Liu, Tiansheng;
Hughes, Michael P;
Peng, Yunan;
McKay, Tristan;
Waddington, Simon N;
... Harvey, Kirsten; + view all
(2024)
Dysregulated Wnt and NFAT signaling in a Parkinson's disease LRRK2 G2019S knock-in model.
Scientific Reports
, 14
(1)
, Article 12393. 10.1038/s41598-024-63130-8.
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2023
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Morfopoulou, Sofia;
Buddle, Sarah;
Montaguth, Oscar Enrique Torres;
Atkinson, Laura;
Guerra-Assunção, José Afonso;
Marjaneh, Mahdi Moradi;
Chiozzi, Riccardo Zenezini;
... Breuer, Judith; + view all
(2023)
Genomic investigations of unexplained acute hepatitis in children.
Nature
10.1038/s41586-023-06003-w.
(In press).
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Ng, Joanne;
Barral, Serena;
Waddington, Simon N;
Kurian, Manju A;
(2023)
Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches.
Cells
, 12
(13)
, Article 1737. 10.3390/cells12131737.
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Ng, Joanne;
Barral, Serena;
Waddington, Simon N;
Kurian, Manju A;
(2023)
Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases.
Movement Disorders
10.1002/mds.29416.
(In press).
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Pauzuolyte, Valda;
Patel, Aara;
Wawrzynski, James R;
Ingham, Neil J;
Leong, Yeh Chwan;
Karda, Rajvinder;
Bitner-Glindzicz, Maria;
... Sowden, Jane C; + view all
(2023)
Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease.
EMBO Molecular Medicine
, Article e17393. 10.15252/emmm.202317393.
(In press).
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Waddington, Simon N;
Peranteau, William H;
Rahim, Ahad A;
Boyle, Ashley K;
Kurian, Manju A;
Gissen, Paul;
Chan, Jerry KY;
(2023)
Fetal gene therapy.
Journal of Inherited Metabolic Disease
10.1002/jimd.12659.
(In press).
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Whiteley, Zoe;
Massaro, Giulia;
Gkogkos, Georgios;
Gavriilidis, Asterios;
Waddington, Simon N;
Rahim, Ahad A;
Craig, Duncan QM;
(2023)
Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors.
Nanoscale
10.1039/d2nr06401d.
(In press).
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2022
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Suleman, S;
Schrubaji, K;
Filippou, C;
Ignatova, S;
Hewitson, P;
Huddleston, J;
Karda, R;
... Themis, M; + view all
(2022)
Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology.
Journal of Virological Methods
, 299
, Article 114305. 10.1016/j.jviromet.2021.114305.
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Suleman, Saqlain;
Payne, Annette;
Bowden, Johnathan;
Haque, Sharmin Al;
Zahn, Marco;
Fawaz, Serena;
Khalifa, Mohammad S;
... Themis, Michael; + view all
(2022)
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival.
Gene Therapy
, 29
pp. 720-729.
10.1038/s41434-022-00335-4.
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2021
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Baruteau, J;
Cunningham, SC;
Yilmaz, BS;
Perocheau, DP;
Eaglestone, S;
Burke, D;
Thrasher, AJ;
... Gissen, P; + view all
(2021)
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.
Molecular Therapy - Methods & Clinical Development
, 23
pp. 135-146.
10.1016/j.omtm.2021.09.005.
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Bates, EA;
Counsell, JR;
Alizert, S;
Baker, AT;
Suff, N;
Boyle, A;
Bradshaw, AC;
... Parker, AL; + view all
(2021)
In vitro and in vivo evaluation of human adenovirus type 49 as a vector for therapeutic applications.
Viruses
, 13
(8)
, Article 1483. 10.3390/v13081483.
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Counsell, JR;
De Brabandere, G;
Karda, R;
Moore, M;
Greco, A;
Bray, A;
Diaz, JA;
... Waddington, SN; + view all
(2021)
Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation.
Molecular Therapy - Methods and Clinical Development
, 20
pp. 357-365.
10.1016/j.omtm.2020.12.005.
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Massaro, G;
Geard, AF;
Liu, W;
Coombe-Tennant, O;
Waddington, SN;
Baruteau, J;
Gissen, P;
(2021)
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Biomolecules
, 11
(4)
, Article 611. 10.3390/biom11040611.
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Ng, J;
Barral, S;
Barrigon, CDLF;
Lignani, G;
Erdem, FA;
Wallings, R;
Privolizzi, R;
... Kurian, MA; + view all
(2021)
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.
Science Translational Medicine
, 13
(594)
, Article eaaw1564. 10.1126/scitranslmed.aaw1564.
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2020
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Boyle, AK;
Suff, N;
Waddington, SN;
Peebles, D;
(2020)
Ascending Vaginal Bacterial Infection with Bioluminescent E. coli Induces Perinatal Neuroinflammation in Mice.
Presented at: 67th Annual Scientific Meeting of the Society for Reproductive Investigation (SRI), Vancouver, Canada.
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Delhove, JM;
Karda, R;
FitzPatrick, LM;
Buckley, SMK;
Waddington, SN;
McKay, TR;
(2020)
Non-invasive somatotransgenic bioimaging in living animals [version 1; peer review: awaiting peer review].
F1000Research
, 9
, Article 1216. 10.12688/f1000research.25274.1.
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Karda, R;
Rahim, AA;
Wong, AMS;
Suff, N;
Diaz, JA;
Perocheau, DP;
Tijani, M;
... Waddington, SN; + view all
(2020)
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
Scientific Reports
, 10
(1)
, Article 2121. 10.1038/s41598-020-59075-3.
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Massaro, G;
Hughes, MP;
Whaler, SM;
Wallom, K-L;
Priestman, DA;
Platt, FM;
Waddington, SN;
(2020)
Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes.
Human Molecular Genetics
10.1093/hmg/ddz317.
(In press).
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Plotegher, N;
Perocheau, D;
Ferrazza, R;
Massaro, G;
Bhosale, G;
Zambon, F;
Rahim, AA;
... Duchen, MR; + view all
(2020)
Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.
Cell Death & Differentiation
, 27
pp. 1588-1603.
10.1038/s41418-019-0442-2.
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Sagar, R;
Almeida-Porada, G;
Blakemore, K;
Chan, JKY;
Choolani, M;
Götherström, C;
Jaulent, A;
... David, AL; + view all
(2020)
Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement.
Molecular Therapy
, 28
(11)
pp. 2316-2319.
10.1016/j.ymthe.2020.10.012.
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Santos, C;
Pai, YJ;
Mahmood, MR;
Leung, K-Y;
Savery, D;
Waddington, SN;
Copp, AJ;
(2020)
Impaired folate one-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase-deficient mice.
Journal of Clinical Investigation
, 130
(3)
pp. 1446-1452.
10.1172/jci132360.
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Soria, LR;
Gurung, S;
De Sabbata, G;
Perocheau, D;
De Angelis, A;
Bruno, G;
Polishchuk, E;
... Brunetti-Pierri, N; + view all
(2020)
Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders.
EMBO Molecular Medicine
, Article e13158. 10.15252/emmm.202013158.
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Suff, N;
Rajvinder, K;
Diaz, A;
Ng, J;
Baruteau, J;
Perocheau, D;
Taylor, P;
... Peebles, DM; + view all
(2020)
Cervical gene delivery of the antimicrobial peptide, Human β‐defensin (HBD)-3, in a mouse model of ascending infection-related preterm birth.
Frontiers in Immunology
, 11
, Article 106. 10.3389/fimmu.2020.00106.
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2019
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Al-Allaf, FA;
Abduljaleel, Z;
Athar, M;
Taher, MM;
Khan, W;
Mehmet, H;
Colakogullari, M;
... Bouazzaoui, A; + view all
(2019)
Modifying inter-cistronic sequence significantly enhances IRES dependent second gene expression in bicistronic vector: Construction of optimised cassette for gene therapy of familial hypercholesterolemia.
Non-coding RNA Research
, 4
(1)
pp. 1-14.
10.1016/j.ncrna.2018.11.005.
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Almeida-Porada, G;
Waddington, SN;
Chan, JKY;
Peranteau, WH;
MacKenzie, T;
Porada, CD;
(2019)
In Utero Gene Therapy Consensus Statement from the IFeTIS.
[Editorial comment].
Molecular Therapy
, 27
(4)
pp. 705-707.
10.1016/j.ymthe.2019.02.015.
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Baruteau, J;
Khalil, Y;
Grunewald, S;
Zancolli, M;
Chakrapani, A;
Cleary, M;
Davison, J;
... Mills, P; + view all
(2019)
Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment.
Metabolites
, 9
(11)
, Article 275. 10.3390/metabo9110275.
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Baruteau, J;
Waddington, SN;
(2019)
Fetal gene therapy for neurodegenerative lysosomal storage diseases.
Journal of Inherited Metabolic Disease
, 42
(3)
pp. 391-393.
10.1002/jimd.12018.
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Chan, JKY;
Gil-Farina, I;
Johana, N;
Rosales, C;
Tan, YW;
Ceiler, J;
Mcintosh, J;
... Mattar, CNZ; + view all
(2019)
Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.
FASEB Journal
, 33
(3)
pp. 3954-3967.
10.1096/fj.201801391R.
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Diaz, JA;
Geard, A;
FitzPatrick, LM;
Delhove, JMKM;
Buckley, SMK;
Waddington, SN;
McKay, TR;
(2019)
Continual Conscious Bioluminescent Imaging in Freely Moving Mice.
Methods in Molecular Biology
, 2081
pp. 161-175.
10.1007/978-1-4939-9940-8_11.
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Karda, R;
Counsell, JR;
Karbowniczek, K;
Caproni, LJ;
Tite, JP;
Waddington, N;
(2019)
Production of lentiviral vectors using novel, enzymatically-produced, linear DNA vectors.
Gene Therapy
, 26
pp. 86-92.
10.1038/s41434-018-0056-1.
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Perocheau, D;
Cunningham, S;
Lee, J;
Antinao Diaz, J;
Waddington, SN;
Gilmour, K;
Eaglestone, S;
... Baruteau, J; + view all
(2019)
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.
Human Gene Therapy
, 30
(1)
pp. 79-87.
10.1089/hum.2018.098.
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Shangaris, P;
Loukogeorgakis, SP;
Subramaniam, S;
Flouri, C;
Jackson, LH;
Wang, W;
Blundell, MP;
... David, AL; + view all
(2019)
Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
[Corrigendum].
Scientific Reports
, 9
, Article 20214. 10.1038/s41598-019-55754-y.
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Shangaris, P;
Loukogeorgakis, S;
Subramaniam, S;
Flouri, C;
Jackson, L;
WANG, W;
Blundell, M;
... David, A; + view all
(2019)
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
Scientific Reports
, 9
, Article 11592. 10.1038/s41598-019-48078-4.
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2018
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Ahmed, SG;
Waddington, SN;
Boza-Morán, MG;
Yáñez-Muñoz, RJ;
(2018)
High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors.
Journal of Controlled Release
, 273
pp. 99-107.
10.1016/j.jconrel.2017.12.029.
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Baruteau, J;
Perocheau, DP;
Hanley, J;
Lorvellec, M;
Rocha-Ferreira, E;
Karda, R;
Ng, J;
... Waddington, SN; + view all
(2018)
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.
Nature Communications
, 9
(1)
, Article 3505. 10.1038/s41467-018-05972-1.
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Counsell, JR;
Karda, R;
Diaz, JA;
Carey, L;
Wiktorowicz, T;
Buckley, SMK;
Ameri, S;
... Howe, SJ; + view all
(2018)
Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.
Molecular Therapy - Nucleic Acids
, 12
pp. 626-634.
10.1016/j.omtn.2018.07.006.
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Dighe, NM;
Tan, KW;
Tan, LG;
Shaw, SSW;
Buckley, SMK;
Sandikin, D;
Johana, N;
... Mattar, CNZ; + view all
(2018)
A comparison of intrauterine haemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassaemia in a HbbTh3/+ murine model.
Experimental Hematology
, 62
10.1016/j.exphem.2018.03.006.
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Edey, LF;
Georgiou, H;
O'Dea, KP;
Mesiano, S;
Herbert, BR;
Lei, K;
Hua, R;
... Johnson, MR; + view all
(2018)
Progesterone, the maternal immune system and the onset of parturition in the mouse.
Biology of Reproduction
, 98
(3)
pp. 376-395.
10.1093/biolre/iox146.
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FitzPatrick, LM;
Hawkins, KE;
Delhove, JMKM;
Fernandez, E;
Soldati, C;
Bullen, LF;
Nohturfft, A;
... McKay, TR; + view all
(2018)
NF-κB Activity Initiates Human ESC-Derived Neural Progenitor Cell Differentiation by Inducing a Metabolic Maturation Program.
Stem Cell Reports
10.1016/j.stemcr.2018.03.015.
(In press).
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Hughes, MP;
Smith, DA;
Morris, L;
Fletcher, C;
Colaco, A;
Huebecker, M;
Tordo, J;
... Rahim, AA; + view all
(2018)
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.
Human Molecular Genetics
, 27
(17)
pp. 3079-3098.
10.1093/hmg/ddy212.
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Massaro, G;
Mattar, CNZ;
Wong, AMS;
Sirka, E;
Buckley, SMK;
Herbert, BR;
Karlsson, S;
... Rahim, AA; + view all
(2018)
Fetal gene therapy for neurodegenerative disease of infants.
Nature Medicine
, 24
pp. 1317-1323.
10.1038/s41591-018-0106-7.
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Massaro, Giulia;
(2018)
Intravenously administered gene therapy for neuronopathic Gaucher disease.
Doctoral thesis (Ph.D), UCL (University College London).
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Suff, N;
Karda, R;
Antinao Diaz, J;
Ng, J;
Baruteau, J;
Perocheau, D;
Tangney, M;
... Waddington, SN; + view all
(2018)
Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth and Neonatal Brain Injury in Pregnant Mice.
American Journal of Pathology
, 188
(10)
pp. 2164-2176.
10.1016/j.ajpath.2018.06.016.
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Tordo, J;
O'Leary, C;
Antunes, ASLM;
Palomar, N;
Aldrin-Kirk, P;
Basche, M;
Bennett, A;
... Henckaerts, E; + view all
(2018)
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Brain
10.1093/brain/awy126.
(In press).
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2017
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Alonso-Ferrero, ME;
van Til, NP;
Bartolovic, K;
Mata, MF;
Wagemaker, G;
Moulding, D;
Williams, DA;
... Howe, SJ; + view all
(2017)
Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors.
Exp Hematol
10.1016/j.exphem.2017.09.003.
(In press).
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Baruteau, J;
Jameson, E;
Morris, AA;
Chakrapani, A;
Santra, S;
Vijay, S;
Kocadag, H;
... Davison, JE; + view all
(2017)
Expanding the phenotype in argininosuccinic aciduria: need for new therapies.
Journal of Inherited Metabolic Disease
, 40
(3)
pp. 357-368.
10.1007/s10545-017-0022-x.
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Baruteau, J;
Waddington, SN;
Alexander, IE;
Gissen, P;
(2017)
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.
Journal of Inherited Metabolic Disease
, 40
(4)
pp. 497-517.
10.1007/s10545-017-0053-3.
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Baruteau, J;
Waddington, SN;
Alexander, IE;
Gissen, P;
(2017)
Delivering efficient liver-directed AAV-mediated gene therapy.
Gene Therapy
, 24
(5)
pp. 263-264.
10.1038/gt.2016.90.
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Baruteau, JC;
(2017)
A gene therapy approach for Argininosuccinic aciduria.
Doctoral thesis , UCL (University College London).
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Counsell, JR;
Asgarian, Z;
Meng, J;
Ferrer, V;
Vink, CA;
Howe, SJ;
Waddington, SN;
... Danos, O; + view all
(2017)
Lentiviral vectors can be used for full-length dystrophin gene therapy.
Scientific Reports
, 7
(1)
, Article 79. 10.1038/s41598-017-00152-5.
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Counsell, JR;
Asgarian, Z;
Meng, J;
Ferrer, V;
Vink, CA;
Howe, SJ;
Waddington, SN;
... Danos, O; + view all
(2017)
Lentiviral vectors can be used for full-length dystrophin gene therapy.
Scientific Reports
, 7
, Article 44775. 10.1038/srep44775.
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Delhove, JM;
Buckley, SM;
Perocheau, DP;
Karda, R;
Arbuthnot, P;
Henderson, NC;
Waddington, SN;
(2017)
Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice.
Scientific Reports
, 7
, Article 41874. 10.1038/srep41874.
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Hanley, J;
Kumar Dhar, D;
Mazzacuva, F;
Fiadeiro, R;
Burden, JJ;
Lyne, AM;
Smith, H;
... Gissen, P; + view all
(2017)
Vps33b is Crucial for Structural and Functional Hepatocyte Polarity.
Journal of Hepatology
, 66
(5)
pp. 1001-1011.
10.1016/j.jhep.2017.01.001.
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Karda, R;
Perocheau, DP;
Suff, N;
Ng, J;
Delhove, JMKM;
Buckley, SMK;
Richards, S;
... Waddington, SN; + view all
(2017)
Continual conscious bioluminescent imaging in freely moving somatotransgenic mice.
Sci Rep
, 7
(1)
, Article 6374. 10.1038/s41598-017-06696-w.
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Mattar, CNZ;
Gil-Farina, I;
Rosales, C;
Johana, N;
Tan, YYW;
McIntosh, J;
Kaeppel, C;
... Chan, JKY; + view all
(2017)
In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.
Molecular Therapy
, 25
(8)
pp. 1843-1853.
10.1016/j.ymthe.2017.04.003.
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Suff, N;
Waddington, SN;
(2017)
The power of bioluminescence imaging in understanding host-pathogen interactions.
Methods
, 127
pp. 69-78.
10.1016/j.ymeth.2017.07.001.
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Vink, CA;
Counsell, JR;
Perocheau, DP;
Karda, R;
Buckley, SMK;
Brugman, MH;
Galla, M;
... Howe, SJ; + view all
(2017)
Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.
Molecular Therapy
, 25
(8)
pp. 1790-1804.
10.1016/j.ymthe.2017.04.028.
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2016
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Banushi, B;
Forneris, F;
Straatman-Iwanowska, A;
Strange, A;
Lyne, A-M;
Rogerson, C;
Burden, JJ;
... Gissen, P; + view all
(2016)
Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis.
Nature Communications
, 7
, Article 12111. 10.1038/ncomms12111.
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Buckinx, R;
Van Remoortel, S;
Gijsbers, R;
Waddington, SN;
Timmermans, JP;
(2016)
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