Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

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http://www.wikidata.org/entity/Q35224393
Duchenne Muscular Dystrophy: From Diagnosis to TherapyDystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophiesAnti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factorsIn vitro stability of therapeutically relevant, internally truncated dystrophins.piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.2'-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping.Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach.Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.Effective regeneration of dystrophic muscle using autologous iPSC-derived progenitors with CRISPR-Cas9 mediated precise correction.Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy.Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions.Skipping Multiple Exons to Treat DMD-Promises and Challenges.
P2860

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

Item
http://www.wikidata.org/entity/Q35224393
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2015 nî lūn-bûn
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2015年論文
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2015年論文
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2015年論文
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Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
@ast
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
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type
label
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
@ast
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
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prefLabel
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
@ast
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
@en
P1433
P1476
P2093
P2860
P304
P31
P356
P433
P478
P50
P577
P5875
P698
P932
P356
P1433
P1476
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45
@en
P2093
P2860
P304
P31
P356
P433
P478
P50
P577
P5875
P698
P932