Zobrazeno 1 - 10
of 45
pro vyhledávání: '"Dany P Perocheau"'
Autor:
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
Publikováno v:
EMBO Molecular Medicine, Vol 13, Iss 2, Pp 1-14 (2020)
Abstract Urea cycle disorders (UCD) are inherited defects in clearance of waste nitrogen with high morbidity and mortality. Novel and more effective therapies for UCD are needed. Studies in mice with constitutive activation of autophagy unravelled Be
Externí odkaz:
https://doaj.org/article/19085dda865a4f648e70bb866bfbf0bc
Autor:
Adrian Westhaus, Marti Cabanes-Creus, Kimberley L. Dilworth, Erhua Zhu, David Salas Gómez, Renina G. Navarro, Anais K. Amaya, Suzanne Scott, Magdalena Kwiatek, Alexandra L. McCorkindale, Tara E. Hayman, Silke Frahm, Dany P. Perocheau, Bang Manh Tran, Elizabeth Vincan, Sharon L. Wong, Shafagh A. Waters, Georgina E. Riddiough, Marcos V. Perini, Laurence O.W. Wilson, Julien Baruteau, Sebastian Diecke, Gloria González-Aseguinolaza, Giorgia Santilli, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
The liver is a prime target for in vivo gene therapies using recombinant adeno-associated viral vectors (rAAV). Multiple clinical trials have been undertaken for this target in the past 15 years, however we are still to see market approval of the fir
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::456eba301b4b73f9427328cdc937aa92
http://edoc.mdc-berlin.de/23212/1/23212oa.pdf
http://edoc.mdc-berlin.de/23212/1/23212oa.pdf
Autor:
Sharon C. Cunningham, Julien Baruteau, Derek Burke, Adrian J. Thrasher, Berna Seker Yilmaz, Simon Eaglestone, Paul Gissen, Simon N. Waddington, Ian E. Alexander, Dany P. Perocheau, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 135-146 (2021)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
X-linked inherited ornithine transcarbamylase deficiency (OTCD) is the most common disorder affecting the liver-based urea cycle, a pathway enabling detoxification of nitrogen waste and endogenous arginine biosynthesis. Patients develop acute hyperam
Autor:
Adrian Westhaus, Marti Cabanes-Creus, Kimberley L. Dilworth, Erhua Zhu, David Salas Gómez, Renina G. Navarro, Anais K. Amaya, Suzanne Scott, Magdalena Kwiatek, Alexandra L. McCorkindale, Tara E. Hayman, Silke Frahm, Dany P. Perocheau, Bang Manh Tran, Elizabeth Vincan, Sharon L. Wong, Shafagh A. Waters, Laurence O. W. Wilson, Julien Baruteau, Sebastian Diecke, Gloria González-Aseguinolaza, Giorgia Santilli, Adrian J. Thrasher, Ian E. Alexander, Leszek Lisowski
The liver is a prime target for in vivo gene therapies using recombinant adeno-associated viral vectors (rAAV). Multiple clinical trials have been undertaken for this target in the past 15 years, however we are still to see market approval of the fir
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ae8acdce92d8cb8bbee102c1127b55a1
https://doi.org/10.1101/2022.09.28.510021
https://doi.org/10.1101/2022.09.28.510021
Autor:
Ulrike Mock, Juan Antinao Diaz, Simon N. Waddington, Dany P. Perocheau, Marc Moore, John R. Counsell, Rajvinder Karda, Antonio Greco, Alysha Bray, Guillaume De Brabandere
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 357-365 (2021)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Lentiviral (LV) vectors based on human immunodeficiency virus type I (HIV-1) package two copies of their single-stranded RNA into vector particles. Normally, this RNA genome is reverse transcribed into a double-stranded DNA provirus that integrates i
Autor:
Tristan R. McKay, Julien Baruteau, Jonathan D. Cooper, Rajvinder Karda, John R. Counsell, Els Henckaerts, Juan Antinao Diaz, Ahad A. Rahim, Simon N. Waddington, Dany P. Perocheau, Nuria Palomar Martin, Maha Tijani, Andrew Wong, Michael P. Hughes, Joanne Ng, Juliette M. K. M. Delhove, Suzanne M. K. Buckley, Natalie Suff
Publikováno v:
Scientific Reports, Vol 10, Iss 1, Pp 1-9 (2020)
Scientific Reports
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 M K M, 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 ', Scientific Reports, vol. 10, no. 1, 2121 . https://doi.org/10.1038/s41598-020-59075-3
Scientific Reports
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 M K M, 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 ', Scientific Reports, vol. 10, no. 1, 2121 . https://doi.org/10.1038/s41598-020-59075-3
We have previously designed a library of lentiviral vectors to generate somatic-transgenic rodents to monitor signalling pathways in diseased organs using whole-body bioluminescence imaging, in conscious, freely moving rodents. We have now expanded t
Autor:
Dany P. Perocheau, Gyorgy Szabadkai, Giulia Massaro, Ruggero Ferrazza, Nicoletta Plotegher, Gauri Bhosale, Michael R. Duchen, Ahad A. Rahim, Graziano Guella, Federico Zambon, Simon N. Waddington
Publikováno v:
Cell Death and Differentiation
Heterozygous mutations of the lysosomal enzyme glucocerebrosidase (GBA1) represent the major genetic risk for Parkinson’s disease (PD), while homozygous GBA1 mutations cause Gaucher disease, a lysosomal storage disorder, which may involve severe ne
Pharmacological modulation of T cell immunity results in long-term remission of autoimmune arthritis
Autor:
Yi Shu Huang, Louise M. Topping, Peter Ericsson, Trevor W. Stone, Felix I.L. Clanchy, Anette Sundstedt, Wen Yi Tseng, Nien Yi Chiang, Zhong Tian Xue, Hans Olov Sjögren, Kay McNamee, Leif G. Salford, Hsi-Hsien Lin, Shue Fen Luo, Dany P. Perocheau, Joy Ogbechi, Richard O. Williams
Publikováno v:
Proc Natl Acad Sci U S A
Chronic inflammatory diseases like rheumatoid arthritis are characterized by a deficit in fully functional regulatory T cells. DNA-methylation inhibitors have previously been shown to promote regulatory T cell responses and, in the present study, we
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ea36dc466bbf5a3277448705b0da3cbd
https://doi.org/10.1073/pnas.2100939118
https://doi.org/10.1073/pnas.2100939118
Publikováno v:
British Journal of Pharmacology
Exosomes are a subset of extracellular vesicles essential for cell-cell communication in health and disease with the ability to transport nucleic acids, functional proteins and other metabolites. Their clinical use as diagnostic biomarkers and therap
Autor:
Simon N. Waddington, Julien Baruteau, Andrea Motta, Gemma Bruno, Carmine Settembre, Youssef Khalil, Andrés F. Muro, Simon Eaton, Leandro R. Soria, Giulia De Sabbata, Elena Polishchuk, Michael Orford, Nicola Brunetti-Pierri, Debora Paris, Sonam Gurung, Philippa B. Mills, Dany P. Perocheau, Paola Cuomo, Angela De Angelis
Publikováno v:
EMBO molecular medicine
e13158 (2021). doi:10.15252/emmm.202013158
info:cnr-pdr/source/autori:Soria L.R.; Gurung S.; De Sabbata G.; Perocheau D.P.; De Angelis A.; Bruno G.; Polishchuk E.; Paris D.; Cuomo P.; Motta A.; Orford M.; Khalil Y.; Eaton S.; Mills P.B.; Waddington S.N.; Settembre C.; Muro A.F.; Baruteau J.; Brunetti-Pierri N./titolo:Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders/doi:10.15252%2Femmm.202013158/rivista:EMBO molecular medicine (Print)/anno:2021/pagina_da:/pagina_a:/intervallo_pagine:/volume:e13158
EMBO Molecular Medicine
EMBO Molecular Medicine, Vol 13, Iss 2, Pp n/a-n/a (2021)
e13158 (2021). doi:10.15252/emmm.202013158
info:cnr-pdr/source/autori:Soria L.R.; Gurung S.; De Sabbata G.; Perocheau D.P.; De Angelis A.; Bruno G.; Polishchuk E.; Paris D.; Cuomo P.; Motta A.; Orford M.; Khalil Y.; Eaton S.; Mills P.B.; Waddington S.N.; Settembre C.; Muro A.F.; Baruteau J.; Brunetti-Pierri N./titolo:Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders/doi:10.15252%2Femmm.202013158/rivista:EMBO molecular medicine (Print)/anno:2021/pagina_da:/pagina_a:/intervallo_pagine:/volume:e13158
EMBO Molecular Medicine
EMBO Molecular Medicine, Vol 13, Iss 2, Pp n/a-n/a (2021)
Urea cycle disorders (UCD) are inherited defects in clearance of waste nitrogen with high morbidity and mortality. Novel and more effective therapies for UCD are needed. Studies in mice with constitutive activation of autophagy unravelled Beclin‐1