Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A

Autor: Suzan Boutary, Marie Caillaud, Mévidette El Madani, Jean-Michel Vallat, Julien Loisel-Duwattez, Alice Rouyer, Laurence Richard, Céline Gracia, Giorgia Urbinati, Didier Desmaële, Andoni Echaniz-Laguna, David Adams, Patrick Couvreur, Michael Schumacher, Charbel Massaad, Liliane Massaad-Massade

Publikováno v: Communications Biology, Vol 4, Iss 1, Pp 1-14 (2021)

Boutary et al. describe siRNA based therapy conjugated with squalene nanoparticles as an efficient approach to normalize PMP22 protein levels, restore locomotor activity, electrophysiological parameters and function of myelin sheath in CMT1A mouse mo

Externí odkaz: https://doaj.org/article/1040014b286640ea9f55ad4f4445c8c9

Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A

Autor: Jean-Michel Vallat, Didier Desmaële, Laurence Richard, Céline Gracia, Julien Loisel-Duwattez, Marie Caillaud, Mévidette El Madani, David Adams, Alice Rouyer, Liliane Massaad-Massade, Patrick Couvreur, Michael Schumacher, Giorgia Urbinati, Andoni Echaniz-Laguna, Suzan Boutary, Charbel Massaad

Publikováno v: Communications Biology
Communications Biology, Nature Publishing Group, 2021, 4 (1), ⟨10.1038/s42003-021-01839-2⟩
Communications Biology, Vol 4, Iss 1, Pp 1-14 (2021)
Communications Biology, 2021, 4 (1), pp.317. ⟨10.1038/s42003-021-01839-2⟩

Charcot-Marie-Tooth disease type 1 A (CMT1A) lacks an effective treatment. We provide a therapy for CMT1A, based on siRNA conjugated to squalene nanoparticles (siRNA PMP22-SQ NPs). Their administration resulted in normalization of Pmp22 protein level

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::00ba7e1ad7fba100a835e29b9666018f
https://hal.archives-ouvertes.fr/hal-03189470/document

Newly identified LMO3-BORCS5 fusion oncogene in Ewing sarcoma at relapse is a driver of tumor progression

Autor: Célia, Dupain, Céline, Gracia, Anne C, Harttrampf, Julie, Rivière, Birgit, Geoerger, Liliane, Massaad-Massade

Publikováno v: Oncogene. 38(47)

Recently, we detected a new fusion transcript LMO3-BORCS5 in a patient with Ewing sarcoma within a cohort of relapsed pediatric cancers. LMO3-BORCS5 was as highly expressed as the characteristic fusion oncogene EWS/FLI1. However, the expression level

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::786f06d2b0fa869073baecda67418973
https://pubmed.ncbi.nlm.nih.gov/31488873

The small RNA SraG participates in PNPase homeostasis

Autor: Mathieu Ballouche, Joël Caillet, Fanette Fontaine, Céline Gracia, Eliane Hajnsdorf, Antonin Marchais, Elise Gasiorowski

Publikováno v: RNA. 22:1560-1573

The rpsO-pnp operon encodes ribosomal protein S15 and polynucleotide phosphorylase, a major 3′–5′ exoribonuclease involved in mRNA decay in Escherichia coli. The gene for the SraG small RNA is located between the coding regions of the rpsO and

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c39ab39f3fa1b9c996183a79f758d823
https://doi.org/10.1261/rna.055236.115

Clostridium difficile Hfq can replace Escherichia coli Hfq for most of its function

Autor: Fanette Fontaine, Céline Gracia, Joël Caillet, Eliane Hajnsdorf

Publikováno v: RNA
RNA, Cold Spring Harbor Laboratory Press, 2014, 20, pp.1567-1578

A gene for the Hfq protein is present in the majority of sequenced bacterial genomes. Its characteristic hexameric ring-like core structure is formed by the highly conserved N-terminal regions. In contrast, the C-terminal forms an extension, which va

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fbcc330101554c63a82f4d6e2e72847e
https://doi.org/10.1261/rna.043372.113