Hasil Pencarian - Simon N Waddington

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  1. 1
    Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches

    Dopamine Transporter Deficiency Syndrome (DTDS): Expanding the Clinical Phenotype and Precision Medicine Approaches oleh Joanne Ng, Serena Barral, Simon N. Waddington, Manju A. Kurian

    Diterbitkan 2023-06-01
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  2. 2
    Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth

    Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth oleh Natalie Suff, Natalie Suff, Natalie Suff, Rajvinder Karda, Juan Antinao Diaz, Joanne Ng, Julien Baruteau, Julien Baruteau, Dany Perocheau, Peter W. Taylor, Dagmar Alber, Suzanne M. K. Buckley, Mona Bajaj-Elliott, Mona Bajaj-Elliott, Simon N. Waddington, Simon N. Waddington, Donald Peebles

    Diterbitkan 2020-02-01
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  3. 3
    Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

    Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development oleh Giulia Massaro, Amy F. Geard, Wenfei Liu, Oliver Coombe-Tennant, Simon N. Waddington, Julien Baruteau, Paul Gissen, Ahad A. Rahim

    Diterbitkan 2021-04-01
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  4. 4
    Perinatal systemic gene delivery using adeno-associated viral vectors

    Perinatal systemic gene delivery using adeno-associated viral vectors oleh Rajvinder eKarda, Suzanne M K Buckley, Citra N Mattar, Joanne eNg, Giulia eMassaro, Michael eHughes, Manju A Kurian, Julien eBaruteau, Paul eGissen, Jerry K Y Chan, Chiara eBacchelli, Simon N Waddington, Simon N Waddington, Ahad A Rahim

    Diterbitkan 2014-11-01
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  5. 5
    Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment

    Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment oleh Julien Baruteau, Youssef Khalil, Stephanie Grunewald, Marta Zancolli, Anupam Chakrapani, Maureen Cleary, James Davison, Emma Footitt, Simon N. Waddington, Paul Gissen, Philippa Mills

    Diterbitkan 2019-11-01
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  6. 6
    Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery

    Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery oleh Megha S. Nivsarkar, Suzanne M. K. Buckley, Alan L. Parker, Dany Perocheau, Tristan R. McKay, Ahad A. Rahim, Steven J. Howe, Simon N. Waddington

    Diterbitkan 2015-01-01
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  7. 7
    Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model

    Dysregulated Wnt and NFAT signaling in a Parkinson’s disease LRRK2 G2019S knock-in model oleh Andrea Wetzel, Si Hang Lei, Tiansheng Liu, Michael P. Hughes, Yunan Peng, Tristan McKay, Simon N. Waddington, Simone Grannò, Ahad A. Rahim, Kirsten Harvey

    Diterbitkan 2024-05-01
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  8. 8
    Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation

    Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation oleh John R. Counsell, Guillaume De Brabandere, Rajvinder Karda, Marc Moore, Antonio Greco, Alysha Bray, Juan Antinao Diaz, Dany P. Perocheau, Ulrike Mock, Simon N. Waddington

    Diterbitkan 2021-03-01
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  9. 9
    Regionally-specified second trimester fetal neural stem cells reveals differential neurogenic programming.

    Regionally-specified second trimester fetal neural stem cells reveals differential neurogenic programming. oleh Yiping Fan, Guillaume Marcy, Eddy S M Lee, Steve Rozen, Citra N Z Mattar, Simon N Waddington, Eyleen L K Goh, Mahesh Choolani, Jerry K Y Chan

    Diterbitkan 2014-01-01
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  10. 10
    Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

    Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys oleh Julien Baruteau, Sharon C. Cunningham, Berna Seker Yilmaz, Dany P. Perocheau, Simon Eaglestone, Derek Burke, Adrian J. Thrasher, Simon N. Waddington, Leszek Lisowski, Ian E. Alexander, Paul Gissen

    Diterbitkan 2021-12-01
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  11. 11
    Lentiviral vectors can be used for full-length dystrophin gene therapy

    Lentiviral vectors can be used for full-length dystrophin gene therapy oleh John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos

    Diterbitkan 2017-03-01
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  12. 12
    Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease

    Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease oleh Valda Pauzuolyte, Aara Patel, James R Wawrzynski, Neil J Ingham, Yeh Chwan Leong, Rajvinder Karda, Maria Bitner‐Glindzicz, Wolfgang Berger, Simon N Waddington, Karen P Steel, Jane C Sowden

    Diterbitkan 2023-10-01
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  13. 13
    In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications

    In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications oleh Emily A. Bates, John R. Counsell, Sophie Alizert, Alexander T. Baker, Natalie Suff, Ashley Boyle, Angela C. Bradshaw, Simon N. Waddington, Stuart A. Nicklin, Andrew H. Baker, Alan L. Parker

    Diterbitkan 2021-07-01
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  14. 14
    Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome

    Human HPSE2 gene transfer ameliorates bladder pathophysiology in a mutant mouse model of urofacial syndrome oleh Filipa M Lopes, Celine Grenier, Benjamin W Jarvis, Sara Al Mahdy, Adrian Lène-McKay, Alison M Gurney, William G Newman, Simon N Waddington, Adrian S Woolf, Neil A Roberts

    Diterbitkan 2024-07-01
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  15. 15
    Continual conscious bioluminescent imaging in freely moving somatotransgenic mice

    Continual conscious bioluminescent imaging in freely moving somatotransgenic mice oleh Rajvinder Karda, Dany P. Perocheau, Natalie Suff, Joanne Ng, Juliette M. K. M. Delhove, Suzanne M. K. Buckley, Samantha Richards, John R. Counsell, Henrik Hagberg, Mark R. Johnson, Tristan R. McKay, Simon N. Waddington

    Diterbitkan 2017-07-01
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  16. 16
    NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming

    NRF2 Orchestrates the Metabolic Shift during Induced Pluripotent Stem Cell Reprogramming oleh Kate E. Hawkins, Shona Joy, Juliette M.K.M. Delhove, Vassilios N. Kotiadis, Emilio Fernandez, Lorna M. Fitzpatrick, James R. Whiteford, Peter J. King, Juan P. Bolanos, Michael R. Duchen, Simon N. Waddington, Tristan R. McKay

    Diterbitkan 2016-03-01
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  17. 17
    Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders

    Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders oleh Leandro R Soria, Sonam Gurung, Giulia De Sabbata, Dany P Perocheau, Angela De Angelis, Gemma Bruno, Elena Polishchuk, Debora Paris, Paola Cuomo, Andrea Motta, Michael Orford, Youssef Khalil, Simon Eaton, Philippa B Mills, Simon N Waddington, Carmine Settembre, Andrés F Muro, Julien Baruteau, Nicola Brunetti‐Pierri

    Diterbitkan 2021-02-01
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  18. 18
    Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice

    Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice oleh John R. Counsell, Rajvinder Karda, Juan Antinao Diaz, Louise Carey, Tatiana Wiktorowicz, Suzanne M.K. Buckley, Shima Ameri, Joanne Ng, Julien Baruteau, Filipa Almeida, Rohan de Silva, Roberto Simone, Eleonora Lugarà, Gabriele Lignani, Dirk Lindemann, Axel Rethwilm, Ahad A. Rahim, Simon N. Waddington, Steven J. Howe

    Diterbitkan 2018-09-01
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  19. 19
    Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

    Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer oleh Julien Baruteau, Dany P. Perocheau, Joanna Hanley, Maëlle Lorvellec, Eridan Rocha-Ferreira, Rajvinder Karda, Joanne Ng, Natalie Suff, Juan Antinao Diaz, Ahad A. Rahim, Michael P. Hughes, Blerida Banushi, Helen Prunty, Mariya Hristova, Deborah A. Ridout, Alex Virasami, Simon Heales, Stewen J. Howe, Suzanne M. K. Buckley, Philippa B. Mills, Paul Gissen, Simon N. Waddington

    Diterbitkan 2018-08-01
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  20. 20
    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

    Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Us... oleh Panicos Shangaris, Stavros P. Loukogeorgakis, Sindhu Subramaniam, Christina Flouri, Laurence H. Jackson, Wei Wang, Michael P. Blundell, Shanrun Liu, Simon Eaton, Nahla Bakhamis, Durrgah Latchumi Ramachandra, Panayiotis Maghsoudlou, Luca Urbani, Simon N. Waddington, Ayad Eddaoudi, Joy Archer, Michael N. Antoniou, Daniel J. Stuckey, Manfred Schmidt, Adrian J. Thrasher, Thomas M. Ryan, Paolo De Coppi, Anna L. David

    Diterbitkan 2019-12-01
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