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A combinantion treatment of SMA cells with LBH589 and an antisense oligonucleotide that mimic Nusinersen (ASO_ISSN1) produced additive effects on SMN2 splicing and SMN protein expression (Pagliarini et al., 2019)

The most well-known is megaloblastic anaemia, a condition in which red blood cells become abnormally large and cannot mature properly, thereby impairing their ability to carry oxygen

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