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Correction to: Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies

The Original Article was published on 20 May 2021

Correction to: Translational Neurodegeneration (2021) 10:16

Following publication of the original article [1], the authors would like to correct a formula from “T > C” to “C > T” in two paragraphs.

  1. 1.

    In the third paragraph of the section Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), the correct sentence should be:

However, the synonymous C > T substitution in SMN2 exon 7 alters an exonic splicing enhancer into an exonic splicing silencer, which predominantly leads to an unstable transcript missing exon 7.

  1. 2.

    In the fourth paragraph of the section Splice-switching AOs, the correct sentence should be:

The C > T substitution in SMN2 creates an exon-splicing silencer and leads to the omission of exon 7 and an unstable SMN protein that is subject to rapid ubiquitinproteasome degradation.

In addition, the authors identified an error in Fig. 4. The correct figure is given below:

Fig. 4
figure 4

Milestones of the development of antisense oligonucleotide therapeutics (excluding siRNA) from bench to bedside. Approved drugs in red are splice-switching antisense oligomers. AO: antisense oligonucleotides; FDA: US Food and Drug Administration; CMV: cytomegalovirus retinitis (in immunocompromised patients); HoFH: Homozygous familial hypercholesterolemia; DMD: Duchenne muscular dystrophy; SMA: spinal muscular atrophy; HTA: Hereditary transthyretin-mediated amyloidosis; BD: Batten disease

The original article [1] has been corrected.


  1. Li, et al. Transl Neurodegener. 2021;10:16.

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Correspondence to May Thandar Aung-Htut.

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Li, D., McIntosh, C.S., Mastaglia, F.L. et al. Correction to: Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies. Transl Neurodegener 10, 41 (2021).

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