From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides.
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GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe DiseaseEuropean consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.Deep intronic mutations and human disease.The humanistic burden of Pompe disease: are there still unmet needs? A systematic review.Glycogen Reduction in Myotubes of Late-Onset Pompe Disease Patients Using Antisense Technology.Novel NTRK1 mutations in Chinese patients with congenital insensitivity to pain with anhidrosis.
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P2860
From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides.
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2016 nî lūn-bûn
@nan
2016年の論文
@ja
2016年論文
@yue
2016年論文
@zh-hant
2016年論文
@zh-hk
2016年論文
@zh-mo
2016年論文
@zh-tw
2016年论文
@wuu
2016年论文
@zh
2016年论文
@zh-cn
name
From Cryptic Toward Canonical ...... of Antisense Oligonucleotides.
@en
type
label
From Cryptic Toward Canonical ...... of Antisense Oligonucleotides.
@en
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From Cryptic Toward Canonical ...... of Antisense Oligonucleotides.
@en
P2093
P2860
P356
P1476
From Cryptic Toward Canonical ...... of Antisense Oligonucleotides.
@en
P2093
Ans T van der Ploeg
Atze J Bergsma
Frans W Verheijen
Stijn Lm In 't Groen
W W M Pim Pijnappel
P2860
P356
10.1038/MTNA.2016.75
P577
2016-09-13T00:00:00Z