Zobrazeno 1 - 10
of 15
pro vyhledávání: '"Peggy Sanatine"'
Autor:
Amelie Raby, Sonia Missiroli, Peggy Sanatine, Dominique Langui, Julien Pansiot, Nissai Beaude, Lucie Vezzana, Rachelle Saleh, Martina Marinello, Mireille Laforge, Paolo Pinton, Ana Buj-Bello, Andrea Burgo
Publikováno v:
iScience, Vol 27, Iss 9, Pp 110683- (2024)
Summary: Mitochondria-endoplasmic reticulum (ER) contact sites (MERCs) emerged to play critical roles in numerous cellular processes, and their dysregulation has been associated to neurodegenerative disorders. Mutations in the SPG4 gene coding for sp
Externí odkaz:
https://doaj.org/article/7c8d77dc77d541b690d64de75d52c7d1
Autor:
Laure Chatrousse, Thifaine Poullion, Lina El-Kassar, Karine Giraud-Triboult, Claire Boissart, Peggy Sanatine, Peter Sommer, Alexandra Benchoua
Publikováno v:
Stem Cell Research, Vol 72, Iss , Pp 103209- (2023)
Phelan-McDermid syndrome (PMS) is a rare genetic disease characterized by a global developmental delay with autism spectrum disorder. PMS is caused by loss of function mutations in the SHANK3 gene leading to SHANK3 protein haploinsufficiency. This st
Externí odkaz:
https://doaj.org/article/c9b85207ac214bd8b6e8596d2804dd53
Autor:
Aude Parcelier, Julien Buisset, Emilie Neveu, Bastien Kauffmann, Sandrine Fraboulet, Peggy Sanatine, Samia Martin, C?drick Rousseaux, Nadia Avenier, Sabine Charrier
Publikováno v:
Re:GEN Open, Vol 2, Iss 1, Pp 28-36 (2022)
Background: In the context of ex vivo gene therapy or chimeric antigen receptor T cell (CAR-T) cell therapy, vector copy number (VCN) analysis in transduced cells by lentiviral vectors enables the assessment of risk and therapeutic efficiency in pati
Externí odkaz:
https://doaj.org/article/1891b6ea0fe24f2fbad1c22dc45dcd7f
Autor:
Jérôme Poupiot, Helena Costa Verdera, Romain Hardet, Pasqualina Colella, Fanny Collaud, Laurent Bartolo, Jean Davoust, Peggy Sanatine, Federico Mingozzi, Isabelle Richard, Giuseppe Ronzitti
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 83-100 (2019)
The pro-tolerogenic environment of the liver makes this tissue an ideal target for gene replacement strategies. In other peripheral tissues such as the skeletal muscle, anti-transgene immune response can result in partial or complete clearance of the
Externí odkaz:
https://doaj.org/article/6845d6f4cd904df4a534435ae9208607
Autor:
Lionel Galibert, Aurélien Jacob, Adrien Savy, Yohann Dickx, Delphine Bonnin, Christophe Lecomte, Lise Rivollet, Peggy Sanatine, Marjorie Boutin Fontaine, Christine Le Bec, Otto-Wilhelm Merten
Publikováno v:
Microorganisms, Vol 9, Iss 9, p 1799 (2021)
Large-scale manufacturing of rAAV is a bottleneck for the development of genetic disease treatments. The baculovirus/Sf9 cell system underpins the first rAAV treatment approved by EMA and remains one of the most advanced platforms for rAAV manufactur
Externí odkaz:
https://doaj.org/article/4577bcd650f74a5eb809f151a65eb0f0
Autor:
Elena Barbon, Mattia Ferrarese, Laetitia van Wittenberghe, Peggy Sanatine, Giuseppe Ronzitti, Fanny Collaud, Pasqualina Colella, Mirko Pinotti, Federico Mingozzi
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 5, Iss C (2016)
Disease-causing splicing mutations can be rescued by variants of the U1 small nuclear RNA (U1snRNAs). However, the evaluation of the efficacy and safety of modified U1snRNAs as therapeutic tools is limited by the availability of cellular and animal m
Externí odkaz:
https://doaj.org/article/4c15efae9b224fc59c9992b12979aab5
Autor:
Daniel Stockholm, Frédérique Edom-Vovard, Sophie Coutant, Peggy Sanatine, Yoshiaki Yamagata, Guillaume Corre, Laurent Le Guillou, Thi My Anh Neildez-Nguyen, Andràs Pàldi
Publikováno v:
PLoS ONE, Vol 5, Iss 12, p e14441 (2010)
BACKGROUND: In culture, isogenic mammalian cells typically display enduring phenotypic heterogeneity that arises from fluctuations of gene expression and other intracellular processes. This diversity is not just simple noise but has biological releva
Externí odkaz:
https://doaj.org/article/ce338b04a5e345839f26ad4545577deb
Autor:
Ai Vu Hong, Nathalie Bourg, Peggy Sanatine, Jerome Poupiot, Karine Charton, Evelyne Gicquel, Emmanuelle Massourides, Marco Spinazzi, Isabelle Richard, David Israeli
Publikováno v:
Life Science Alliance. 6:e202201506
Duchenne muscular dystrophy (DMD) is a severe muscle disease caused by impaired expression of dystrophin. Whereas mitochondrial dysfunction is thought to play an important role in DMD, the mechanism of this dysfunction remains to be clarified. Here w
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1dc4e2c13bcd27d64ace2f777cfcaef6
https://doi.org/10.26508/lsa.202201506
https://doi.org/10.26508/lsa.202201506
Autor:
Laurent Bartolo, Jean Davoust, Helena Costa Verdera, Romain Hardet, Jerome Poupiot, Giuseppe Ronzitti, Isabelle Richard, Peggy Sanatine, Pasqualina Colella, Federico Mingozzi, Fanny Collaud
Publikováno v:
Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2019, 15, pp.83-100. ⟨10.1016/j.omtm.2019.08.012⟩
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss, Pp 83-100 (2019)
Molecular Therapy-Methods and Clinical Development, 2019, 15, pp.83-100. ⟨10.1016/j.omtm.2019.08.012⟩
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2019, 15, pp.83-100. ⟨10.1016/j.omtm.2019.08.012⟩
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss, Pp 83-100 (2019)
Molecular Therapy-Methods and Clinical Development, 2019, 15, pp.83-100. ⟨10.1016/j.omtm.2019.08.012⟩
Molecular Therapy. Methods & Clinical Development
The pro-tolerogenic environment of the liver makes this tissue an ideal target for gene replacement strategies. In other peripheral tissues such as the skeletal muscle, anti-transgene immune response can result in partial or complete clearance of the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0497b7f5010e99b21637b46756974918
https://hal.archives-ouvertes.fr/hal-02392771
https://hal.archives-ouvertes.fr/hal-02392771
Autor:
Mirko Pinotti, Mattia Ferrarese, Pasqualina Colella, Fanny Collaud, Giuseppe Ronzitti, Laetitia van Wittenberghe, Peggy Sanatine, Federico Mingozzi, Elena Barbon
Publikováno v:
Molecular Therapy-Nucleic Acids
Molecular Therapy-Nucleic Acids, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩
Molecular Therapy. Nucleic Acids
Molecular Therapy-Nucleic Acids, Elsevier, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩
Molecular Therapy: Nucleic Acids, Vol 5, Iss C (2016)
Molecular Therapy-Nucleic Acids, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩
Molecular Therapy. Nucleic Acids
Molecular Therapy-Nucleic Acids, Elsevier, 2016, 5, pp.e392. ⟨10.1038/mtna.2016.97⟩
Molecular Therapy: Nucleic Acids, Vol 5, Iss C (2016)
International audience; Disease-causing splicing mutations can be rescued by variants of the U1 small nuclear RNA (U1snRNAs). However, the evaluation of the efficacy and safety of modified U1snRNAs as therapeutic tools is limited by the availability
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3717d2bdc43a1a36b6698c7a7b43a941
https://hal.sorbonne-universite.fr/hal-01449506/document
https://hal.sorbonne-universite.fr/hal-01449506/document