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Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive disease affecting ∼1 in 10,000 live births. The most striking component is the loss of α-motor neurons in the ventral horn of the spinal cord, resulting in progressive paralysis and eventually premature death. There is no current treatment para...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
मुख्य लेखकों: Porensky, Paul N., Burghes, Arthur H.M.
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: Mary Ann Liebert, Inc. 2013
विषय:
ऑनलाइन पहुंच:https://ncbi.nlm.nih.gov/pmc/articles/PMC3655628/
https://ncbi.nlm.nih.gov/pubmed/23544870
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.225
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