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2009 | 1 |
2011 | 2 |
2025 | 0 |
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Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.
Nature. 2011 Oct 5;478(7367):123-6. doi: 10.1038/nature10485.
Nature. 2011.
PMID: 21979052
Free PMC article.
Splicing therapeutics in SMN2 and APOB.
Khoo B, Krainer AR.
Khoo B, et al.
Curr Opin Mol Ther. 2009 Apr;11(2):108-15.
Curr Opin Mol Ther. 2009.
PMID: 19330716
Free PMC article.
Review.
Item in Clipboard
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.
Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH.
Passini MA, et al.
Sci Transl Med. 2011 Mar 2;3(72):72ra18. doi: 10.1126/scitranslmed.3001777.
Sci Transl Med. 2011.
PMID: 21368223
Free PMC article.
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