Zanetta, C (1 January 2014). Molecular Therapeutic Strategies for Spinal Muscular Atrophies: Current and Future Clinical Trials. Clinical Therapeutics36 (1): 128–140. PMID24360800. DOI: 10.1016/j.clinthera.2013.11.006.
Peng Wen Pao, Keng Boon Wee, Woon Chee Yee and Zacharias Aloysius DwiPramono (April 2014). Dual Masking of Specific Negative Splicing Regulatory Elements Resulted in Maximal Exon 7 Inclusion of SMN2 Gene. Molecular Therapy22: 854–861. PMID24317636. PMC3982506. DOI: 10.1038/mt.2013.276. The sequence of nusinersen (UCACUUUCAUAAUGCUGG) is listed as N1-Hua in Table I.
Garber, K (11 October 2016). Big win possible for Ionis/Biogen antisense drug in muscular atrophy.. Nature Biotechnology34 (10): 1002–1003. PMID27727217. DOI: 10.1038/nbt1016-1002.
Zanetta, C (1 January 2014). Molecular Therapeutic Strategies for Spinal Muscular Atrophies: Current and Future Clinical Trials. Clinical Therapeutics36 (1): 128–140. PMID24360800. DOI: 10.1016/j.clinthera.2013.11.006.
Peng Wen Pao, Keng Boon Wee, Woon Chee Yee and Zacharias Aloysius DwiPramono (April 2014). Dual Masking of Specific Negative Splicing Regulatory Elements Resulted in Maximal Exon 7 Inclusion of SMN2 Gene. Molecular Therapy22: 854–861. PMID24317636. PMC3982506. DOI: 10.1038/mt.2013.276. The sequence of nusinersen (UCACUUUCAUAAUGCUGG) is listed as N1-Hua in Table I.
Garber, K (11 October 2016). Big win possible for Ionis/Biogen antisense drug in muscular atrophy.. Nature Biotechnology34 (10): 1002–1003. PMID27727217. DOI: 10.1038/nbt1016-1002.