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Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment

Spinal muscular atrophy (SMA) is the most common genetic disease affecting infants and young adults. Due to mutation/deletion of the survival motor neuron (SMN) gene, SMA is characterized by the SMN protein lack, resulting in motor neuron impairment, skeletal muscle atrophy and premature death. Even...

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Vydáno v:Front Pharmacol
Hlavní autoři: Menduti, Giovanna, Rasà, Daniela Maria, Stanga, Serena, Boido, Marina
Médium: Artigo
Jazyk:Inglês
Vydáno: Frontiers Media S.A. 2020
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7689316/
https://ncbi.nlm.nih.gov/pubmed/33281605
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fphar.2020.592234
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