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Items: 5

1.

Generation and Characterization of a genetic zebrafish model of SMA carrying the human SMN2 gene.

Hao le T, Burghes AH, Beattie CE.

Mol Neurodegener. 2011 Mar 28;6(1):24. doi: 10.1186/1750-1326-6-24.

2.

Therapeutics development for spinal muscular atrophy.

Sumner CJ.

NeuroRx. 2006 Apr;3(2):235-45. Review.

3.

Spinal muscular atrophy: present state.

Schmalbruch H, Haase G.

Brain Pathol. 2001 Apr;11(2):231-47. Review.

PMID:
11303798
4.

[The role of RNA splicing in the pathogenesis of spinal muscular atrophy and development of its therapeutics].

Sahashi K, Sobue G.

Brain Nerve. 2014 Dec;66(12):1471-80. doi: 10.11477/mf.1416200061. Review. Japanese.

PMID:
25475034
5.

Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy.

Jablonka S, Sendtner M.

Gene Ther. 2017 May 30. doi: 10.1038/gt.2017.46. [Epub ahead of print] Review.

PMID:
28556834

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