Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency.

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http://www.wikidata.org/entity/Q46849509

description

2011 nî lūn-bûn

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2011年の論文

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2011年学术文章

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2011年学术文章

@zh-cn

2011年学术文章

@zh-hans

2011年学术文章

@zh-my

2011年学术文章

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2011年學術文章

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2011年學術文章

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2011年學術文章

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name

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

@en

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

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type

Item

label

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

@en

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

@nl

prefLabel

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

@en

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

@nl

P1476

Pseudoexon exclusion by antise ...... dropterin synthase deficiency.

@en

P2093

Anahita Rassi

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Beat Thöny

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Belen Pérez

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David Meili

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Hiu Man Viecelli

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Lourdes R Desviat

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Magdalena Ugarte

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Sandra Brasil

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P2860

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients

Disorders of tetrahydrobiopterin metabolism and their treatment

Disorders of biopterin metabolism

Fatal propionic acidemia in mice lacking propionyl-CoA carboxylase and its rescue by postnatal, liver-specific supplementation via a transgene

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.

Design and synthesis of dendritic molecular transporter that achieves efficient in vivo delivery of morpholino antisense oligo.

Transvascular delivery of small interfering RNA to the central nervous system.

In vivo correction of a Menkes disease model using antisense oligonucleotides.

Modulating the expression of disease genes with RNA-based therapy.

P304

1019-1027

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scholarly article

P356

10.1002/HUMU.21529

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P478

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P577

2011-08-04T00:00:00Z

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P5875

51097846

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21542064

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