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Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy
Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of infant mortality. SMA results from insufficient survival motor neuron (SMN) protein due to alternative splicing. Antisense oligonucleotides, gene therapy and splicing modifiers recently received FDA approval. Al...
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| Pubblicato in: | Life Sci Alliance |
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| Autori principali: | , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Life Science Alliance LLC
2020
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7723287/ https://ncbi.nlm.nih.gov/pubmed/33234679 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.26508/lsa.202000889 |
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