Correction of cilia structure and function alleviates multi-organ pathology in Bardet–Biedl syndrome mice

Bardet–Biedl syndrome (BBS) is a pleiotropic autosomal recessive ciliopathy affecting multiple organs. The development of potential disease-modifying therapy for BBS will require concurrent targeting of multi-systemic manifestations. Here, we show for the first time that monosialodihexosylgangliosid...

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Detalles Bibliográficos
Autores principales: Husson, Hervé, Bukanov, Nikolay O, Moreno, Sarah, Smith, Mandy M, Richards, Brenda, Zhu, Cheng, Picariello, Tyler, Park, Hyejung, Wang, Bing, Natoli, Thomas A, Smith, Laurie A, Zanotti, Stefano, Russo, Ryan J, Madden, Stephen L, Klinger, Katherine W, Modur, Vijay, Ibraghimov-Beskrovnaya, Oxana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
General Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7471507/
https://www.ncbi.nlm.nih.gov/pubmed/32620959
http://dx.doi.org/10.1093/hmg/ddaa138

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