Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

about

Delivery is key: lessons learnt from developing splice-switching antisense therapies Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy.Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy RNA Editing for Muscular Dystrophy Therapy

P2860

Q33625875-D5F2097F-1455-4CE0-B052-5E9395D0ACDB Q52564618-97F41A0D-89C9-496E-A0AD-5C2F3414AD8A Q57039821-919036EC-2C69-49EC-A394-45462F38C9AF Q58051671-53573E81-0034-4F34-9A99-51D369EC021C

P2860

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

Item

http://www.wikidata.org/entity/Q40667121

description

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

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

@en

type

Item

label

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

@en

prefLabel

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

@en

P1476

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

@en

P2093

Amédée Mollard

http://www.w3.org/2001/XMLSchema#string

Anita Le Heron

http://www.w3.org/2001/XMLSchema#string

George Dickson

http://www.w3.org/2001/XMLSchema#string

Graham McClorey

http://www.w3.org/2001/XMLSchema#string

Laura Julien

http://www.w3.org/2001/XMLSchema#string

Maëva Le Hir

http://www.w3.org/2001/XMLSchema#string

Susan Jarmin

http://www.w3.org/2001/XMLSchema#string

Thomas Voit

http://www.w3.org/2001/XMLSchema#string

P2860

Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.

Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

P304

3555-3563

http://www.w3.org/2001/XMLSchema#string

P31

scholarly article

P356

10.1093/HMG/DDW201

http://www.w3.org/2001/XMLSchema#string

P433

http://www.w3.org/2001/XMLSchema#string

P478

http://www.w3.org/2001/XMLSchema#string

P50

Stéphanie Lorain

Sofia Benkhelifa-Ziyyat

Cécile Peccate

France Piétri-Rouxel

Matthew J. Wood

P577

2016-07-04T00:00:00Z

http://www.w3.org/2001/XMLSchema#dateTime

P698

27378686

http://www.w3.org/2001/XMLSchema#string

P921

gene therapy

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

about

P2860

P2860

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.

description

name

type

label

prefLabel

P1433

P1476

P2093

P2860

P304

P31

P356

P433

P478

P50

P577

P698

P921

P356

P1433

P1476

P2093

P2860

P304

P31

P356

P433

P478

P50

P577

P698

P921