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How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy

Spinal muscular atrophy (SMA), a prominent genetic disease of infant mortality, is caused by low levels of survival motor neuron (SMN) protein owing to deletions or mutations of the SMN1 gene. SMN2, a nearly identical copy of SMN1 present in humans, cannot compensate for the loss of SMN1 due to pred...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Veröffentlicht in:Gene Ther
Hauptverfasser: Singh, Natalia N., Howell, Matthew D., Androphy, Elliot J., Singh, Ravindra N.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5623086/
https://ncbi.nlm.nih.gov/pubmed/28485722
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2017.34
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