Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
Author:
Publisher
Oxford University Press (OUP)
Subject
Genetics (clinical),Genetics,Molecular Biology,General Medicine
Link
http://academic.oup.com/hmg/article-pdf/26/R2/R151/20425066/ddx215.pdf
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4. Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy;Hum. Mol. Genet,2008
5. Cardiac defects contribute to the pathology of spinal muscular atrophy models;Hum. Mol. Genet,2010
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