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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...
Autores principales: | , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2020
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Materias: | |
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 |