The exosome journey: from biogenesis to uptake and intracellular signalling

מאת Gurung, Sonam, Perocheau, Dany, Touramanidou, Loukia, Baruteau, Julien
הוצא לאור ב Cell Commun Signal (2021)

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

מאת Baruteau, Julien, Waddington, Simon N., Alexander, Ian E., Gissen, Paul
הוצא לאור ב J Inherit Metab Dis (2017)

Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease

מאת Seker Yilmaz, Berna, Baruteau, Julien, Rahim, Ahad A., Gissen, Paul
הוצא לאור ב Int J Mol Sci (2020)

Pregnancy outcome in Refsum disease: Affected fetuses and children born to an affected mother

מאת Dubot, Patricia, Astudillo, Léonardo, Touati, Guy, Baruteau, Julien, Broué, Pierre, Roche, Sandrine, Sabourdy, Frédérique, Levade, Thierry
הוצא לאור ב JIMD Rep (2019)

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

מאת Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
הוצא לאור ב Biomolecules (2021)

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment

מאת Baruteau, Julien, Khalil, Youssef, Grunewald, Stephanie, Zancolli, Marta, Chakrapani, Anupam, Cleary, Maureen, Davison, James, Footitt, Emma, Waddington, Simon N., Gissen, Paul, Mills, Philippa
הוצא לאור ב Metabolites (2019)

Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice

מאת Suff, Natalie, Karda, Rajvinder, Diaz, Juan A., Ng, Joanne, Baruteau, Julien, Perocheau, Dany, Tangney, Mark, Taylor, Peter W., Peebles, Donald, Buckley, Suzanne M.K., Waddington, Simon N.
הוצא לאור ב Am J Pathol (2018)

Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C

מאת Héron Bénédicte, Valayannopoulos Vassili, Baruteau Julien, Chabrol Brigitte, Ogier Hélène, Latour Philippe, Dobbelaere Dries, Eyer Didier, Labarthe François, Maurey Hélène, Cuisset Jean-Marie, de Villemeur Thierry, Sedel Frédéric, Vanier Marie T
יצא לאור 2012-06-01

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

מאת Perocheau, Dany P., Cunningham, Sharon C., Lee, Juhee, Antinao Diaz, Juan, Waddington, Simon N., Gilmour, Kimberly, Eaglestone, Simon, Lisowski, Leszek, Thrasher, Adrian J., Alexander, Ian E., Gissen, Paul, Baruteau, Julien
הוצא לאור ב Hum Gene Ther (2019)

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

מאת Suff, Natalie, Karda, Rajvinder, Diaz, Juan Antinao, Ng, Joanne, Baruteau, Julien, Perocheau, Dany, Taylor, Peter W., Alber, Dagmar, Buckley, Suzanne M. K., Bajaj-Elliott, Mona, Waddington, Simon N., Peebles, Donald
הוצא לאור ב Front Immunol (2020)

Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C

מאת Héron, Bénédicte, Valayannopoulos, Vassili, Baruteau, Julien, Chabrol, Brigitte, Ogier, Hélène, Latour, Philippe, Dobbelaere, Dries, Eyer, Didier, Labarthe, François, Maurey, Hélène, Cuisset, Jean-Marie, de Villemeur, Thierry Billette, Sedel, Frédéric, Vanier, Marie T
יצא לאור 2012

Perinatal systemic gene delivery using adeno-associated viral vectors

מאת Karda, Rajvinder, Buckley, Suzanne M. K., Mattar, Citra N., Ng, Joanne, Massaro, Giulia, Hughes, Michael P., Kurian, Manju A., Baruteau, Julien, Gissen, Paul, Chan, Jerry K. Y., Bacchelli, Chiara, Waddington, Simon N., Rahim, Ahad A.
הוצא לאור ב Front Mol Neurosci (2014)

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice

מאת Counsell, John R., Karda, Rajvinder, Diaz, Juan Antinao, Carey, Louise, Wiktorowicz, Tatiana, Buckley, Suzanne M.K., Ameri, Shima, Ng, Joanne, Baruteau, Julien, Almeida, Filipa, de Silva, Rohan, Simone, Roberto, Lugarà, Eleonora, Lignani, Gabriele, Lindemann, Dirk, Rethwilm, Axel, Rahim, Ahad A., Waddington, Simon N., Howe, Steven J.
הוצא לאור ב Mol Ther Nucleic Acids (2018)

Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders

מאת Soria, Leandro R, Gurung, Sonam, De Sabbata, Giulia, Perocheau, Dany P, De Angelis, Angela, Bruno, Gemma, Polishchuk, Elena, Paris, Debora, Cuomo, Paola, Motta, Andrea, Orford, Michael, Khalil, Youssef, Eaton, Simon, Mills, Philippa B, Waddington, Simon N, Settembre, Carmine, Muro, Andrés F, Baruteau, Julien, Brunetti‐Pierri, Nicola
הוצא לאור ב EMBO Mol Med (2020)

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

מאת Karda, Rajvinder, Rahim, Ahad A., Wong, Andrew M. S., Suff, Natalie, Diaz, Juan Antinao, Perocheau, Dany P., Tijani, Maha, Ng, Joanne, Baruteau, Julien, Martin, Nuria Palomar, Hughes, Michael, Delhove, Juliette M. K. M., Counsell, John R., Cooper, Jonathan D., Henckaerts, Els, Mckay, Tristan R., Buckley, Suzanne M. K., Waddington, Simon N.
הוצא לאור ב Sci Rep (2020)

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

מאת Baruteau, Julien, Perocheau, Dany P., Hanley, Joanna, Lorvellec, Maëlle, Rocha-Ferreira, Eridan, Karda, Rajvinder, Ng, Joanne, Suff, Natalie, Diaz, Juan Antinao, Rahim, Ahad A., Hughes, Michael P., Banushi, Blerida, Prunty, Helen, Hristova, Mariya, Ridout, Deborah A., Virasami, Alex, Heales, Simon, Howe, Stewen J., Buckley, Suzanne M. K., Mills, Philippa B., Gissen, Paul, Waddington, Simon N.
הוצא לאור ב Nat Commun (2018)

Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study

מאת Valayannopoulos, Vassili, Baruteau, Julien, Delgado, Maria Bueno, Cano, Aline, Couce, Maria L., Del Toro, Mireia, Donati, Maria Alice, Garcia-Cazorla, Angeles, Gil-Ortega, David, Gomez-de Quero, Pedro, Guffon, Nathalie, Hofstede, Floris C., Kalkan-Ucar, Sema, Coker, Mahmut, Lama-More, Rosa, Martinez-Pardo Casanova, Mercedes, Molina, Agustin, Pichard, Samia, Papadia, Francesco, Rosello, Patricia, Plisson, Celine, Le Mouhaer, Jeannie, Chakrapani, Anupam
הוצא לאור ב Orphanet J Rare Dis (2016)

Expanding the phenotype in argininosuccinic aciduria: need for new therapies

מאת Baruteau, Julien, Jameson, Elisabeth, Morris, Andrew A., Chakrapani, Anupam, Santra, Saikat, Vijay, Suresh, Kocadag, Huriye, Beesley, Clare E., Grunewald, Stephanie, Murphy, Elaine, Cleary, Maureen, Mundy, Helen, Abulhoul, Lara, Broomfield, Alexander, Lachmann, Robin, Rahman, Yusof, Robinson, Peter H., MacPherson, Lesley, Foster, Katharine, Chong, W. Kling, Ridout, Deborah A., Bounford, Kirsten McKay, Waddington, Simon N., Mills, Philippa B., Gissen, Paul, Davison, James E.
הוצא לאור ב J Inherit Metab Dis (2017)

mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria

מאת Gurung, Sonam, Timmermand, Oskar Vilhelmsson, Perocheau, Dany, Gil-Martinez, Ana Luisa, Minnion, Magdalena, Touramanidou, Loukia, Fang, Sherry, Messina, Martina, Khalil, Youssef, Spiewak, Justyna, Barber, Abigail R., Edwards, Richard S., Pinto, Patricia Lipari, Finn, Patrick F., Cavedon, Alex, Siddiqui, Summar, Rice, Lisa, Martini, Paolo G.V., Ridout, Deborah, Heywood, Wendy, Hargreaves, Ian, Heales, Simon, Mills, Philippa B., Waddington, Simon N., Gissen, Paul, Eaton, Simon, Ryten, Mina, Feelisch, Martin, Frassetto, Andrea, Witney, Timothy H., Baruteau, Julien
הוצא לאור ב Sci Transl Med (2024)

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

מאת Ng, Joanne, Barral, Serena, De La Fuente Barrigon, Carmen, Lignani, Gabriele, Erdem, Fatma A., Wallings, Rebecca, Privolizzi, Riccardo, Rossignoli, Giada, Alrashidi, Haya, Heasman, Sonja, Meyer, Esther, Ngoh, Adeline, Pope, Simon, Karda, Rajvinder, Perocheau, Dany, Baruteau, Julien, Suff, Natalie, Diaz, Juan Antinao, Schorge, Stephanie, Vowles, Jane, Marshall, Lucy R., Cowley, Sally A., Sucic, Sonja, Freissmuth, Michael, Counsell, John R., Wade-Martins, Richard, Heales, Simon J.R., Rahim, Ahad A., Bencze, Maximilien, Waddington, Simon N., Kurian, Manju A.
הוצא לאור ב Sci Transl Med (2021)