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Current Challenges in Understanding the Cellular and Molecular Mechanisms in Niemann–Pick Disease Type C1

Rare diseases are a heterogeneous group of very different clinical syndromes. Their most common causes are defects in the hereditary material, and they can therefore be passed on to descendants. Rare diseases become manifest in almost all organs and often have a systemic expressivity, i.e., they aff...

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Dettagli Bibliografici
Pubblicato in:Int J Mol Sci
Autori principali: Bräuer, Anja U., Kuhla, Angela, Holzmann, Carsten, Wree, Andreas, Witt, Martin
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6771135/
https://ncbi.nlm.nih.gov/pubmed/31500175
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20184392
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