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Selected Publications

2023

Whiteley Z, Massaro G, Gkogkos G, Gavriilidis A, Waddington SN, Rahim AA, Craig DQ. Microfluidic production of nanogels as alternative triple transfection reagents for the manufacture of adeno-associated virus vectors. Nanoscale. 2023:15(12):5865-76. https://doi.org/10.1039/D2NR06401D

Morfopoulou S, Buddle S, Montaguth OE, Atkinson L, Guerra-Assunção JA, Marjaneh MM, Chiozzi RZ, Storey N, Campos L, Hutchinson JC, Counsell JR. Genomic investigations of unexplained acute hepatitis in children. Nature. 2023 30:1-2. https://doi.org/10.1038/s41586-023-06003-w

Ng J, Barral S, Waddington SN, Kurian MA. Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases. Movement Disorders. 2023.  https://doi.org/10.1002/mds.29416

Kairuz D, Samudh N, Ely A, Arbuthnot P, Bloom K. Production, Characterization, and Assessment of Permanently Cationic and Ionizable Lipid Nanoparticles for Use in the Delivery of Self-Amplifying RNA Vaccines. Pharmaceutics. 2023, 7;15(4):1173. https://doi.org/10.3390/pharmaceutics15041173

Kana BD, Arbuthnot P, Botwe BK, Choonara YE, Hassan F, Louzir H, Matsoso P, Moore PL, Muhairwe A, Naidoo K, Ndomondo-Sigonda M. Opportunities and challenges of leveraging COVID-19 vaccine innovation and technologies for developing sustainable vaccine manufacturing capabilities in Africa. The Lancet Infectious Diseases. 2023. https://doi.org/10.1016/S1473-3099(22)00878-7

2022

Farhad T, Neves K, Arbuthnot P, Maepa MB. Adenoviral Vectors: Potential as Anti-HBV Vaccines and Therapeutics. Genes. 2022; 13(11):1941. https://doi.org/10.3390/genes13111941

Samudh N, Shrilall C, Arbuthnot P, Bloom K, Ely A. Diversity of Dysregulated Long Non-Coding RNAs in HBV-Related Hepatocellular Carcinoma. Frontiers in Immunology. 2022:129. https://doi.org/10.3389/fimmu.2022.834650

Maepa MB, Ely A, Kramvis A, Bloom K, Naidoo K, Simani OE, Maponga TG, Arbuthnot P. Hepatitis B Virus Research in South Africa. Viruses: 2022:14(9):1939. https://doi.org/10.3390/v14091939

Suleman, Saqlain, Annette Payne, Johnathan Bowden, Sharmin Al Haque, Marco Zahn, Serena Fawaz, Mohammad S. Khalifa et al. HIV-1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival. Gene Therapy:2022: 1-10. https://doi.org/10.1038/s41434-022-00335-4

Jacobs, Ridhwaanah, Prashika Singh, Tiffany Smith, Patrick Arbuthnot, and Mohube Betty Maepa. Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases. Gene Therapy :2022: 1-8. https://doi.org/10.1038/s41434-022-00342-5

Kairuz D, Samudh N, Ely A, Arbuthnot P, Bloom K. Advancing mRNA technologies for therapies and vaccines: An African context. Frontiers in Immunology. 2022:13. https://doi.org/10.3389/fimmu.2022.1018961

Suleman, Saqlain, Kuteiba Schrubaji, Chrysovalanto Filippou, Svetlana Ignatova, Peter Hewitson, Jonathan Huddleston, Rajvinder Karda, Simon N. Waddington, and Michael Themis. Rapid and inexpensive purification of adenovirus vectors using an optimised aqueous two-phase technology. Journal of Virological Methods 299 2022: 114305. https://doi.org/10.1016/j.jviromet.2021.114305

2021

Smith T, Singh P, Chmielewski KO, Bloom K, Cathomen T, Arbuthnot P, Ely A. Improved Specificity and Safety of Anti-Hepatitis B Virus TALENs Using Obligate Heterodimeric FokI Nuclease Domains. Viruses. 2021; 13(7):1344. https://doi.org/10.3390/v13071344

Mosti L, Langner LM, Chmielewski KO, Arbuthnot P, Alzubi J, Cathomen T.(2021) Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells. Gene Ther. 2021 Feb 1. doi: 10.1038/s41434-021-00220-6. Epub ahead of print.

Maepa MB, Bloom K, Ely A, Arbuthnot P. (2021) Hepatitis B virus: promising drug targets and therapeutic implications. Expert Opin Ther Targets. Apr 19:1-16. doi: 10.1080/14728222.2021.1915990. Epub ahead of print.

Singh P, Kairuz D, Arbuthnot P, Bloom K. (2021) Silencing hepatitis B virus covalently closed circular DNA: The potential of an epigenetic therapy approach. World J Gastroenterol. 27(23):3182-3207. doi: 10.3748/wjg.v27.i23.3182.

Bloom, K., van den Berg, F. & Arbuthnot P. (2021) Self-amplifying RNA vaccines for infectious diseases, Gene Therapy, 28:117-129 doi: 10.1038/s41434-020-00204-y.

Ng J, Barral S, De La Fuente Barrigon C, Lignani G, Erdem FA, Wallings R, Privolizzi R, Rossignoli G, Alrashidi H, Heasman S, Meyer E, Ngoh A, Pope S, Karda R, Perocheau D, Baruteau J, Suff N, Antinao Diaz J, Schorge S, Vowles J, Marshall LR, Cowley SA, Sucic S, Freissmuth M, Counsell JR, Wade-Martins R, Heales SJR, Rahim AA, Bencze M, Waddington SN, Kurian MA. (2021) Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism. Sci Transl Med.13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564.

Massaro G, Geard AF, Liu W, Coombe-Tennant O, Waddington SN, Baruteau J, Gissen P, Rahim AA. (2021) Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development. Biomolecules. 11(4):611. doi: 10.3390/biom11040611.

Ely, A., Singh, P., Smith, T. S., & Arbuthnot, P. (2021). In vitro transcribed mRNA for expression of designer nucleases: Advantages as a novel therapeutic for the management of chronic HBV infection, Adv Drug Deliv Rev. 168:134-146 doi:10.1016/j.addr.2020.05.010

Ely A, Bloom K, Maepa MB, Arbuthnot P. (2021) Recent Update on the Role of Circular RNAs in Hepatocellular Carcinoma. J Hepatocell Carcinoma. 8:1-17. doi: 10.2147/JHC.S268291

Counsell JR, De Brabandere G, Karda R, Moore M, Greco A, Bray A, Diaz JA, Perocheau DP, Mock U, Waddington SN. (2021) Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation. Mol Ther Methods Clin Dev. 20:357-365. doi: 10.1016/j.omtm.2020.12.005. eCollection 2021 Mar 12

Soria LR, Gurung S, De Sabbata G, Perocheau DP, De Angelis A, Bruno G, Polishchuk E, Paris D, Cuomo P, Motta A, Orford M, Khalil Y, Eaton S, Mills PB, Waddington SN, Settembre C, Muro AF, Baruteau J, Brunetti-Pierri N. (2021) Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders. EMBO Mol Med. 13(2):e13158. doi: 10.15252/emmm.202013158.

2020

Van den Berg, F., Limani, S. W., Mnyandu, N., Maepa, M. B., Ely, A. & Arbuthnot P. (2020) Advances with RNAi-based therapy for hepatitis B virus infection, Viruses, 12(8): 851; https://doi.org/10.3390/v12080851

Maepa, M. B., Jacobs, R., van den Berg, F., & Arbuthnot, P. (2020). Recent developments with advancing gene therapy to treat chronic infection with hepatitis B virus. Curr Opin HIV AIDS, 15(3), 200-207. doi:10.1097/COH.0000000000000623

Van den Berg, F., Ely, A. & Arbuthnot, P. (2020) Generating DNA Expression Cassettes Encoding Multimeric Artificial MicroRNA Precursors. Methods Mol Biol. 2115, 185-197. doi: 10.1007/978-1-0716-0290-4_11

Mnyandu, N., Arbuthnot, P. & Maepa, M. B. (2020). In Vivo Delivery of Cassettes Encoding Anti-HBV Primary MicroRNAs Using an Ancestral Adeno-Associated Viral Vector. Methods Mol Biol. 2115, 171-183. doi: 10.1007/978-1-0716-0290-4_10.

Sagar. R,. Almeida-Porada, G., Blakemore, K., Chan, J.K.Y., Choolani, M., Götherström, C., Jaulent, A., MacKenzie, T.C., Mattar, C., Porada, C.D., Peranteau, W.H., Schneider, H., Shaw, S.W., Waddington, S.N., Westgren, M. & David, A.L. (2020) Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement. Mol Ther. 28(11), 2316-2319. doi: 10.1016/j.ymthe.2020.10.012.

Diaz, J.A., Geard, A., FitzPatrick, L.M., Delhove, J.M.K.M., Buckley, S.M.K., Waddington, S.N., McKay, T.R. & Karda, R. (2020) Continual Conscious Bioluminescent Imaging in Freely Moving Mice. Methods Mol Biol. 2081, 161-175. doi: 10.1007/978-1-4939-9940-8_11.

Plotegher, N., Perocheau, D., Ferrazza, R., Massaro, G., Bhosale, G., Zambon, F., Rahim, A. A., Guella, G., Waddington, S.N., Szabadkai, G., & Duchen, M. R. (2020). Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload. Cell Death Differ, 27(5), 1588-1603. doi:10.1038/s41418-019-0442-2

Massaro, G., Hughes, M. P., Whaler, S. M., Wallom, K. L., Priestman, D. A., Platt, F. M., Waddington, S.N., & Rahim, A. A. (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. Hum Mol Genet. doi:10.1093/hmg/ddz317

Karda, R., Rahim, A. A., Wong, A. M. S., Suff, N., Diaz, J. A., Perocheau, D. P., Tijani, M., Ng, J., Baruteau, J., Martin, N. P., Hughes, M., Delhove, J., Counsell, J. R., Cooper, J. D., Henckaerts, E., McKay, T. R., Buckley, S. M. K., & Waddington, S.N. (2020). Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors. Sci Rep, 10(1), 2121. doi:10.1038/s41598-020-59075-3

Suff, N., Karda, R., Diaz, J. A., Ng, J., Baruteau, J., Perocheau, D., Taylor, P. W., Alber, D., Buckley, S. M. K., Bajaj-Elliott, M., Waddington, S.N., & Peebles, D. (2020). Cervical Gene Delivery of the Antimicrobial Peptide, Human beta-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth. Front Immunol, 11, 106. doi:10.3389/fimmu.2020.00106

Santos, C., Pai, Y. J., Mahmood, M. R., Leung, K. Y., Savery, D., Waddington, S.N., Copp, A. J., & Greene, N. (2020). Impaired folate 1-carbon metabolism causes formate-preventable hydrocephalus in glycine decarboxylase-deficient mice. J Clin Invest, 130(3), 1446-1452. doi:10.1172/JCI132360

2019

van Dorsten R, Lambson B, Wibmer C, Weinberg M, Moore P, Morris L. 2019. Neutralization breadth and potency of single-chain variable fragments derived from broadly neutralizing antibodies targeting multiple epitopes on the HIV-1 envelope. J Virol. pii: JVI.01533-19. doi: 10.1128/JVI.01533-19.

Bloom K, Kaldine H, Cathomen T, Mussolino C, Ely A, Arbuthnot P. 2019. Inhibition of replication of hepatitis B virus using transcriptional repressors that target the viral DNA. BMC Infect Dis. 12;19(1):802. doi: 10.1186/s12879-019-4436-y.

Ely A, Bloom K, Maepa M.B, Mahlangu J.N, Pepper M.S, Arbuthnot P. 2019. Gene and Cell Therapy in South Africa: Current Status and Future Prospects. S Afr Med J, 109(8, Suppl.1) ppS13-S17. doi: 10.7196/SAMJ.2019.v109i4b.13819. 

Shangaris P, Loukogeorgakis S, Subramaniam S, Flouri C, Jackson L, Wang W, Blundell M, Liu S, Eaton S, Bakhamis N, Ramachandra D, Maghsoudlou P, Urbani L, Waddington S, Eddaoudi A, Archer J, Antoniou M, Stuckey D, Schmidt M, Thrasher A, Ryan T, Coppi P, David A. 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. Sci Rep. 12;9(1):11592. doi: 10.1038/s41598-019-48078-4.

van den Berg F, Makoah N, Ali S, Scott T, Mapengo R, Mutsvunguma L, Mkhize N, Lambson B, Kgagudi P, Crowther C, Abdool Karim S, Balazs A, Weinberg M, Ely A, Arbuthnot P, Morris L. 2019. AAV-Mediated Expression of Broadly Neutralizing and Vaccine-like Antibodies Targeting the HIV-1 Envelope V2 Region. Mol Ther Methods Clin Dev. 12;14:100-112. doi: 10.1016/j.omtm.2019.06.002.

Pinto D, Scott T, DeMarino C, Pleet M, Vo T, Saifuddin M, Kovalskyy D, Erickson J, Cowen M, Barclay R, Zeng C, Weinberg M, Kashanchi F. 2019. Effect of transcription inhibition and generation of suppressive viral non-coding RNAs. Retrovirology. 29;16(1):13. doi: 10.1186/s12977-019-0475-0.

Arbuthnot P, Ely A, Bloom K. 2019. A convenient method to generate and maintain poly(A)-encoding DNA sequences required for in vitro transcription of mRNA. Biotechniques. 66(1):37-39. doi: 10.2144/btn-2018-0120.

Baruteau J, Waddington S. 2019. Fetal gene therapy for neurodegenerative lysosomal storage diseases. J Inherit Metab Dis. 42(3):391-393. doi: 10.1002/jimd.12018.

Karda R, Counsell J, Karbowniczek K, Caproni L, Tite J, Waddington S. 2019. Production of lentiviral vectors using novel, enzymatically produced, linear DNA. Gene Ther. 26(3-4):86-92. doi: 10.1038/s41434-018-0056-1.

Revill P, Chisari F, Block J, Dandri M, Gehring A, Guo H, Hu J, Kramvis A, Lampertico P, Janssen H, Levrero M, Li W, Liang T, Lim S, Lu F, Penicaud M, Tavis J, Thimme R; Members of the ICE-HBV Working Groups; ICE-HBV Stakeholders Group Chairs; ICE-HBV Senior Advisors, Zoulim F. 2019. A global scientific strategy to cure hepatitis B. Lancet Gastroenterol Hepatol. 4(7):545-558. doi: 10.1016/S2468-1253(19)30119-0.

2018

Bloom, K., Maepa, M.B., Ely, A., Arbuthnot, P. 2018. Gene Therapy for Chronic HBV-Can We Eliminate cccDNA? Genes 9(4) pii: E207. 

Maepa, M.B., Ely, A., Arbuthnot, P. 2018. How successful has targeted RNA interference for hepatic fibrosis been? Expert Opinion on Biological Therapy 18(4): 381-388.

Moyo, B., Bloom, K., Scott, T., Ely, A., Arbuthnot, P. 2018. Advances with using CRISPR/Cas-mediated gene editing to treat infections with hepatitis B virus and hepatitis C virus. Virus Research 244: 311-320.

Counsell J, Karda R, Diaz J, Carey L, Wiktorowicz T, Buckley S, Ameri S, Ng J, Baruteau J, Almeida F, de Silva R, Simone R, Lugarà E, Lignani G, Lindemann D, Rethwilm A, Rahim A, Waddington S, Howe S. 2018. Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice. Mol Ther Nucleic Acids. 7;12:626-634. doi: 10.1016/j.omtn.2018.07.006.

Baruteau J, Perocheau D, Hanley J, Lorvellec M, Rocha-Ferreira E, Karda R, Ng J, Suff N, Diaz J, Rahim A, Hughes M, Banushi B, Prunty H, Hristova M, Ridout D, Virasami A, Heales S, Howe S, Buckley S, Mills P, Gissen P, Waddington S. 2018. Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer. Nat Commun. 29;9(1):3505. doi: 10.1038/s41467-018-05972-1.

Tordo J, O'Leary C, Antunes A, Palomar N, Aldrin-Kirk P, Basche M, Bennett A, D'Souza Z, Gleitz H, Godwin A, Holley R, Parker H, Liao A, Rouse P, Youshani A, Dridi L, Martins C, Levade T, Stacey K, Davis D, Dyer A, Clément N, Björklund T, Ali R, Agbandje-McKenna M, Rahim A, Pshezhetsky A, Waddington S, Linden R, Bigger B, Henckaerts E. 2018. A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency. Brain. 1;141(7):2014-2031. doi: 10.1093/brain/awy126.

2017

Scott, T., Moyo, B., Nicholson, S., Maepa, M.B., Watashi, K., Ely, A., Weinberg, M.S., Arbuthnot, P. 2017. ssAAVs containing cassettes encoding SaCas9 and guides targeting hepatitis B virus inactivate replication of the virus in cultured cells. Scientific Reports 7(1): 7401.

Arbuthnot, P., Maepa, M.B., Ely, A., Pepper, M.S. 2017. The state of gene therapy research in Africa, its significance and implications for the future. Gene Therapy 24(9): 581-589.

Delhove, J.M.K.M., Karda, R., Hawkins, K.E., FitzPatrick, L.M., Waddington, S.N., McKay, T.R. 2017. Bioluminescence Monitoring of Promoter Activity In Vitro and In Vivo. Methods in Moleuclar Biology 1651: 49-64.

Baruteau, J., Waddington, S.N., Alexander, I.E., Gissen, P. 2017. Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. Journal of Inherited Metabolic Disease 40(4): 497-517.

Baruteau, J., Jameson, E., Morris, A.A., Chakrapani, A., Santra, S., Vijay, S., Kocadag, H., Beesley, C.E., Grunewald, S., Murphy, E., Cleary, M., Mundy, H., Abulhoul, L., Broomfield, A., Lachmann, R., Rahman, Y., Robinson, P.H., MacPherson, L., Foster, K., Chong, W.K., Ridout, D.A., Bounford, K.M., Waddington, S.N., Mills, P.B., Gissen, P., Davison, J.E. 2017. Expanding the phenotype in argininosuccinic aciduria: need for new therapies. Journal of Inherited Metabolic Disease 40(3): 357-368.

Delhove, J.M., Buckley, S.M., Perocheau, D.P., Karda, R., Arbuthnot, P., Henderson, N.C., Waddington, S.N., McKay, T.R. 2017. Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice. Scientific Reports 7: 41874.

Bloom, K., Ely, A., Arbuthnot, P. 2017. A T7 Endonuclease I Assay to Detect Talen-Mediated Targeted Mutation of HBV cccDNA. Methods in Molecular Biology 1540: 85-95.

Maepa, B.M., Ely, A., Grayson, W. and Arbuthnot, P. 2017. Sustained Inhibition of HBV Replication In Vivo after Systemic Injection of AAVs Encoding Artificial Antiviral Primary MicroRNAs. Molecular Therapy: Nucleic Acids 7: 190-199.

2016

Lamb, C. and Arbuthnot, P. 2016. Activating the innate immune response to counter chronic hepatitis B virus infection. Expert Opinion on Biological Therapy 16(12): 1517-1527.

Mbatha, L., Chakravorty, S., de Koning, C.B., van Otterlo, W.A.L., Arbuthnot, P., Ariatti, M. and Singh, M. 2016. Spacer Length: A determining factor in the design of galactosyl ligands for hepatoma cell-specific liposomal gene delivery. Current Drug Delivery 13: 935-945.

Bourhill, T., Arbuthnot, P. and Ely, A. 2016. Successful disabling of the 5' UTR of HCV using adeno-associated viral vectors to deliver modular multimeric primary microRNA mimics. Journal of Virological Methods 235: 26-33.

Dreyer, T., Nicholson, S., Ely, A., Arbuthnot, P. and Bloom, K. 2016. Improved antiviral efficacy using TALEN-mediated homology directed recombination to introduce artificial primary miRNAs into DNA of hepatitis B virus. Biochemical and Biophysical Research Communications 478(4): 1563-1568.

Ely, A., Moyo, B. and Arbuthnot, P. 2016. Progress with developing use of gene editing to cure chronic infection with hepatitis B virus. Molecular Therapy 24(4): 671-677.

Moyo, B., Nicholson, S.A. and Arbuthnot, P. 2016. The role of long non-coding RNAs in hepatitis B virus-related hepatocellular carcinoma. Virus Research 212: 103-113.

Crowther, C., Mowa, B. and Arbuthnot, P. 2016. Hepatic delivery of artificial micro RNAs using helper-dependent adenoviral vectors. In: Shum, K. and Rossi, J. Eds. SiRNA delivery methods. Methods in Molecular Biology Volume 1364. New York, Springer Science. pp 249-260.

van den Berg, F.T., Rossi, J.J., Arbuthnot, P. and Weinberg, M.S. 2016. Design of effective primary microRNA mimics with different basal stem conformations. Molecular Therapy - Nucleic Acids 5: 1-12.

Saayman, S.M., Lazar, D.C., Scott, T.A., Hart,.J.R., Takahashi, M., Burnett, J.C., Planelles, V., Morris, K.V. and Weinberg, M.S. 2016. Potent and targeted activation of laten HIV-1 using CRISPR/dCas9 activator complex. Molecular Therapy 24(3): 488-498.

Weinberg, M.S. and Morris, K.V. 2016. Transcriptional gene silencing in humans. Nucleic Acids Research 44(14): 6505-6517.

Saayman, S.M., Ackley, A., Burdach, J., Clemson, M., Gruenert, D.C., Tachikawa, K., Chivukula, P., Weinberg, M.S. and Morris, K.V. 2016. Long non-coding RNA Bgas regulates the cystic fibrosis transmembrane conductance regulator. Molecular Therapy 24(8): 1351-1357.

Scholefield, J. and Weinberg, M.S. 2016. The application of CRISPR-Cas9 technologies and therapies in stem cells. Current Stem Cell Reports 2: 95-103.

Hart, J.R., Weinberg, M.S., Morris, K.V., Roberts, T.C., Janda, K.D., Garner, A.L. and Vogt, P.K. 2016. MINCR is not a MYC-induced lncRNA. PNAS 113(5): E496-E497.

Edey, L.F., O’Dea, K.P., Herbert, B.R., Hua, R., Waddington, S.N., MacIntyre, D.A., Bennett, P.R., Takata, M. and Johnson, M.R. 2016. The local and systemic immune response to intrauterine LPS in the prepartum mouse. Biology of Reproduction 95(6): 1-10.

Waddington, S.N., Privolizzi, R., Karda, R. and O’Neill, H.C. 2016. A broad overview and review of CRISPR-Cas technology and stem cells. Current Stem Cell Reports 2: 9-20.

Hawkins, K.E., Joy, S., Delhove, J.M., Kotiadis, V.N., Fernandez, E., Fitzpatrick, L.M., Whiteford, J.R., King, P.J., Bolanos, J.P., Duchen, M.R., Waddington, S.N. and McKay, T.R. 2016. NRF2 orchestrates the metabolic shift during induced pluripotent stem cell reprogramming. Cell Reports 14(8): 1883-1891.

Migale, R., MacIntyre, D.A., Cacciatore, S., Lee, Y.S., Hagberg, H., Herbert, B.R., Johnson, M.R., Peebles, D., Wadddington, S.N. and Bennett, P.R. 2016. Modeling hormonal and inflammatory contributions to preterm and term labor using uterine temporal transcriptomics. BMC Medicine 14(86): 1-17.

Karda, R., Buckley, S.M.K. and Waddington, S.N. 2016. Gene therapy with Adeno-associated virus for cystic fibrosis. American Journal of Respiratory and Critical Care Medicine 193(3): 234-236.

2015

Arbuthnot, P. 2015. Gene therapy for viral infections. Elsevier Academic Press, London. pp 376.

Marimani, M.D., Ely, A., Buff, M.C.R., Bernhardt, S., Engels, J.W., Scherman, D., Escriou, V. and Arbuthnot, P. 2015. Inhibition of replication of hepatitis B virus in transgenic mice following administration of hepatotropic lipoplexes containing guanidinopropyl-modified siRNAs. Journal of Controlled Release 209: 198-206.

Nicholson, S., Moyo, B. and Arbuthnot, P. 2015. Progress and prospects of engineered sequence-specific DNA modulating technologies for the management of liver diseases. World Journal of Hepatology 7(6): 859-873.

Bloom, K., Ely, A. and Arbuthnot, P. 2015. Recent Advances in Use of Gene Therapy to Treat Hepatitis B Virus Infection. Invited article for special issue entitled Gene Therapy for HIV and Chronic Infections; Advances in Experimental Medicine and Biology 848: 31-49.

Buff, M.C.R., Bernhardt, S. Marimani, M.D. Ely, A. Engels, J.B. and Arbuthnot, P. 2015. Use of guanidinopropyl-modified siRNAs to silence gene expression. Methods in Molecular Biology, 2015, pp 217-249.

Ivacik, D., Ely, A., Ferry, N. and Arbuthnot, P. 2015. Sustained Inhibition of hepatitis B virus replication in vivo using RNAi-activating lentiviruses. Gene Therapy 22(2):163-171.

Bloom, K., Mussolino, C. and Arbuthnot, P. 2015. Transcription activator-like effector (TALE) nucleases and repressor TALEs for antiviral gene therapy. Current Stem Cell Reports 1: 1-8.

Govender, D., Ul Islam, R., De Koning, C., van Otterlo, W., Arbuthnot, P., Ariatti, M. and Singh, M. 2015. Stealth lipoplex decorated with triazole-tethered galactosyl moieties: strongly hepatotropic gene vector. Biotechnology Letters 37(3):567-575.

Mowa, M.B., Crowther, C. and Arbuthnot, P. (2015) Recombinant Adenoviral vectors for hepatotropic delivery of expressed RNAi activators. Methods in Molecular Biology, Invited article, In Press.

Buckinx, R., Van Remoortel, S., Gijsbers, R., Waddington, S.N. and Timmermans, J.P. Proof-of-concept: neonatal intravenous injection of adeno-associated virus results in successful transduction of myenteric and submucosal neurons in the mouse small and large intestine. Neurogastroenterology & Motility 28(2): 299-305.

2014

Mowa, M.B., Crowther, C. Ely, A. and Arbuthnot, P. 2014. Inhibition of hepatitis B virus replication in vivo by helper dependent adenoviral vectors expressing artificial antiHBV pri-miRs from a liver specific Pol II promoter, BioMed Research International (formerly titled Journal of Biomedicine and Biotechnology), Special edition on Physiology to the Pleiotropic Role of RNAs: Prospecting Novel Therapies (PLEIO). 

Arbuthnot, P. and Weinberg, M.S. 2014. Edited Book: Applied RNAi: From Fundamental Research to Therapeutic Applications, Caister Press, ISBN: 978-1-908230-43-0, Editors: Arbuthnot, P. & Weinberg, M.S.

Crowther, C, Mowa, MB, Ely A and Arbuthnot, P. 2014. Inhibition of hepatitis B virus replication in vivo using helper-dependent adenovirus vectors to deliver antiviral RNAi expression cassettes, Antiviral Therapy 19, 363-373.

2013

Bloom, K., Ely, A., Mussolino, C., Cathomen, T. and Arbuthnot, P. 2013. Inactivation of hepatitis B virus replication in cultured cells and in vivo with engineered Transcription Activator-Like Effector Nucleases. Molecular Therapy, 21, 1889-1897.

Marimani, M., Ely, A., Buff, M.C.R., Arbuthnot, P. Engels, J.W. 2013. Inhibition of hepatitis B virus replication in cultured cells and in vivo using 2’-O-guanidinopropyl modified siRNAs. BioOrganic & Med Chem21, 6145-6155.

Marimani, M., Hean, J., Bloom, K., Ely, A and Arbuthnot, P. 2013. Recent advances in developing nucleic acid based hepatitis B virus therapy. Future Microbiology, 8(11), 1489–1504.

Crowther, C, Mowa, MB, Ely A and Arbuthnot, P. 2013. Inhibition of hepatitis B virus replication in vivo using helper-dependent adenovirus vectors to deliver antiviral RNAi expression cassettes. Antiviral Therapy (In Press).

Sewbalas, A., Ul Islam, R., van Otterlo, W.A.L., de Koning C.B. Singh, M., Arbuthnot, P. and Ariatti M. 2013. Enhancement of transfection activity in HEK293 cells by lipoplexes containing cholesterol nitrogen-pivoted aza-crown ethers. Med. Chem. Res., 22, 2561-2569.

2012

Pichard, V., Aubert, D., Boni, S., Battaglia, S., Ivacik, D., Arbuthnot, P. and Ferry, N. 2012. Specific Micro RNA-Regulated TetR-KRAB Transcriptional Control of Transgene Expression in Viral Vector-Transduced Cells. PLOS One, 7(12), e51952.

Green, V. A.  Arbuthnot P. and Weinberg M.S. 2012. Impact of sustained RNAi-mediated suppression of cellular cofactor Tat-SF1 on HIV-1 replication in CD4+ T cells. Virology Journal, 9:272.

Taylor, S.H., Harmse, J., Arbuthnot, P, Van Den Berg, F, Weinberg, M.S. and Rey M.E.C. 2012.  Construction of effective inverted repeat silencing constructs using sodium bisulfite treatment coupled with strand-specific PCR. BioTechniques, 52, 254-262.

Scott, T., Paweska J. T., Arbuthnot, P. and Weinberg M.S. 2012. Pathogenic effects of Rift Valley fever virus NSs gene are alleviated in cultured cells by expressed antiviral short hairpin RNAs, Antiviral Therapy. 17(4):643-56.

Brzezinska, J., D’Onofrio, J., Buff, M.C.R., Hean, J. Ely, A., Marimani, M., Arbuthnot, P. Engels, J.W. 2012. Synthesis of 2’-O-guanidinopropyl-modified nucleoside phosphoramidites and their incorporation into siRNAs targeting hepatitis B virus. BioOrganic & Med Chem, 20: 1594-1606.

Mowa, MB, Crowther, C, Ely A and Arbuthnot, P. 2012. Efficient silencing of hepatitis B virus by helper-dependent adenovirus vector-mediated delivery of antiviral primary micro RNA shuttles. Micro RNA. 1, 19-25.

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