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Mouse Survival Motor Neuron Alleles That Mimic SMN2 Splicing and Are Inducible Rescue Embryonic Lethality Early in Development but Not Late

Spinal muscular atrophy (SMA) is caused by low survival motor neuron (SMN) levels and patients represent a clinical spectrum due primarily to varying copies of the survival motor neuron-2 (SMN2) gene. Patient and animals studies show that disease severity is abrogated as SMN levels increase. Since t...

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Библиографические подробности
Главные авторы: Hammond, Suzan M., Gogliotti, Rocky G., Rao, Vamshi, Beauvais, Ariane, Kothary, Rashmi, DiDonato, Christine J.
Формат: Artigo
Язык:Inglês
Опубликовано: Public Library of Science 2010
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3012126/
https://ncbi.nlm.nih.gov/pubmed/21249120
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0015887
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