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Splice-Switching Therapy for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a genetic disorder with severity ranging from premature death in infants to restricted motor function in adult life. Despite the genetic cause of this disease being known for over twenty years, only recently has a therapy been approved to treat the most severe form o...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Genes (Basel)
Egile Nagusiak: Meijboom, Katharina E., Wood, Matthew J.A., McClorey, Graham
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: MDPI 2017
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC5485525/
https://ncbi.nlm.nih.gov/pubmed/28604635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/genes8060161
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