Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping

Bibliografski detalji
Glavni autori:	Goyenvalle, A, Babbs, A, Powell, D, Kole, R, Fletcher, S, Wilton, S, Davies, K
Format:	Conference item
Izdano:	2010

Slični predmeti

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
od: Goyenvalle, A, i dr.
Izdano: (2010)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
od: Goyenvalle, A, i dr.
Izdano: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
od: Goyenvalle, A, i dr.
Izdano: (2012)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
od: Goyenvalle, A, i dr.
Izdano: (2009)

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
od: Cirak, S, i dr.
Izdano: (2011)

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.
od: Cirak, S, i dr.
Izdano: (2011)

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
od: Yin, HF, i dr.
Izdano: (2016)

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
od: Yokota, T, i dr.
Izdano: (2012)

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
od: Yin, H, i dr.
Izdano: (2009)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
od: Lu, Q, i dr.
Izdano: (2003)

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
od: Lu, Q, i dr.
Izdano: (2004)

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.
od: Aoki, Y, i dr.
Izdano: (2010)

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment
od: Betts, C, i dr.
Izdano: (2018)

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
od: Chwalenia, K, i dr.
Izdano: (2022)

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
od: Goyenvalle, A, i dr.
Izdano: (2010)

Microarray analysis of mdx mice expressing high levels of utrophin: therapeutic implications for dystrophin deficiency.
od: Baban, D, i dr.
Izdano: (2008)

Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice
od: Forand, A, i dr.
Izdano: (2020)

Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO
od: Yin, H, i dr.
Izdano: (2010)

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
od: Betts, C, i dr.
Izdano: (2014)

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
od: Goyenvalle, A, i dr.
Izdano: (2011)

Exon skipping is correlated with exon circularization
od: Kelly, S, i dr.
Izdano: (2015)

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
od: Hanson, B, i dr.
Izdano: (2022)

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
od: Guiraud, S, i dr.
Izdano: (2019)

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene.
od: Wakefield, P, i dr.
Izdano: (2000)

Alternative utrophin mRNAs contribute to phenotypic differences between dystrophin-deficient mice and Duchenne muscular dystrophy
od: Perkins, K, i dr.
Izdano: (2018)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.
od: Yin, H, i dr.
Izdano: (2011)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice
od: Yin, H, i dr.
Izdano: (2011)

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.
od: Kinali, M, i dr.
Izdano: (2009)

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.
od: Goyenvalle, A, i dr.
Izdano: (2009)

The utrophin and dystrophin genes share similarities in genomic structure.
od: Pearce, M, i dr.
Izdano: (1993)

Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
od: Goyenvalle, A, i dr.
Izdano: (2011)

Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
od: Culle, M, i dr.
Izdano: (2001)

Generation of clusters of dystrophin revertant fibers requires muscle regeneration and tests the function of dystrophin/utrophin transgenes
od: Yokota, T, i dr.
Izdano: (2004)

Confirmation by immunogold labeling that utrophin is localised to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle
od: Cullen, M, i dr.
Izdano: (2001)

Fully automated fast-flow synthesis of antisense phosphorodiamidate morpholino oligomers
od: Li, Chengxi, i dr.
Izdano: (2021)

A quantitative study of bioenergetics in skeletal muscle lacking utrophin and dystrophin.
od: Cole, M, i dr.
Izdano: (2002)

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.
od: Aoki, Y, i dr.
Izdano: (2012)

Compensation for dystrophin-deficiency: ADAM12 overexpression in skeletal muscle results in increased alpha 7 integrin, utrophin and associated glycoproteins.
od: Moghadaszadeh, B, i dr.
Izdano: (2003)

Utrophin binds laterally along actin filaments and can couple costameric actin with sarcolemma when overexpressed in dystrophin-deficient muscle.
od: Rybakova, I, i dr.
Izdano: (2002)

ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan.
od: Ishikawa-Sakurai, M, i dr.
Izdano: (2004)