Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD

Bibliografiske detaljer
Main Authors:	Godfrey, C, McClorey, G, Coursindel, T, Betts, C, Hammond, S, Andaloussi, S, Gait, M, Wood, M
Format:	Conference item
Udgivet:	2012

Lignende værker

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.
af: Betts, C, et al.
Udgivet: (2012)

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment
af: Betts, C, et al.
Udgivet: (2018)

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy
af: Blain, A, et al.
Udgivet: (2018)

Expression analysis in multiple muscle groups and serum reveals complexity in the microRNA transcriptome of the mdx mouse with implications for therapy.
af: Roberts, T, et al.
Udgivet: (2012)

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient
af: Saito, T, et al.
Udgivet: (2010)

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
af: Betts, C, et al.
Udgivet: (2014)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.
af: Yin, H, et al.
Udgivet: (2011)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice
af: Yin, H, et al.
Udgivet: (2011)

Cell-penetrating peptide conjugates of steric blocking oligonucleotides as therapeutics for neuromuscular diseases from a historical perspective to current prospects of treatment
af: Gait, M, et al.
Udgivet: (2018)

Exon skipping is correlated with exon circularization
af: Kelly, S, et al.
Udgivet: (2015)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
af: Goyenvalle, A, et al.
Udgivet: (2009)

Peptide-mediated cell and in vivo delivery of antisense oligonucleotides and siRNA
af: Järver, P, et al.
Udgivet: (2012)

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
af: Yin, HF, et al.
Udgivet: (2016)

NOVEL CELL PENETRATING PEPTIDES FOR SKELETAL AND CARDIAC MUSCLE DELIVERY OF PMO ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
af: Hammond, S, et al.
Udgivet: (2011)

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.
af: Goyenvalle, A, et al.
Udgivet: (2009)

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
af: Wang, Q, et al.
Udgivet: (2010)

Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle.
af: Ivanova, G, et al.
Udgivet: (2008)

An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides
af: Baker, YR, et al.
Udgivet: (2022)

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
af: Yin, H, et al.
Udgivet: (2009)

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
af: Chwalenia, K, et al.
Udgivet: (2022)

Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO
af: Yin, H, et al.
Udgivet: (2010)

Second-generation compound for the modulation of utrophin in the therapy of DMD
af: Guiraud, S, et al.
Udgivet: (2015)

CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion
af: Yin, Hao, et al.
Udgivet: (2019)

Cell-penetrating peptides to enhance delivery of Oligonucleotide-based therapeutics
af: McClorey, G, et al.
Udgivet: (2018)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
af: Lu, Q, et al.
Udgivet: (2003)

Novel EGFP reporter cell and mouse models for sensitive imaging and quantification of exon skipping
af: Hara, Y, et al.
Udgivet: (2020)

Transcriptional Gene Silencing: An Epigenetic Myostatin Blockade Strategy
af: Roberts, T, et al.
Udgivet: (2011)

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.
af: Aoki, Y, et al.
Udgivet: (2010)

Analysis of coordinated skipped exon pairs using single molecule sequencing technology
af: Adadey, Asa (Asa Owuraku)
Udgivet: (2015)

Machine learning based CRISPR gRNA design for therapeutic exon skipping
af: Louie, Wilson, et al.
Udgivet: (2022)

New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
af: Aoki, Y, et al.
Udgivet: (2012)

Machine learning based CRISPR gRNA design for therapeutic exon skipping
af: Louie, Wilson,M. Eng.Massachusetts Institute of Technology.
Udgivet: (2020)

Advanced project portfolio management and the PMO : multiplying ROI at warp speed /
af: 180283 Kendall, Gerald I., et al.
Udgivet: (2003)

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
af: Lu, Q, et al.
Udgivet: (2004)

Proteomic evaluation of Pip6a-PMO treatment for myotonic dystrophy type 1
af: Holland, A, et al.
Udgivet: (2018)

LEPIGENETIC MYOSTATIN BLOCKADE
af: Roberts, T, et al.
Udgivet: (2011)

Small RNA-Mediated Epigenetic Myostatin Silencing.
af: Roberts, T, et al.
Udgivet: (2012)

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
af: Goyenvalle, A, et al.
Udgivet: (2010)

Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
af: Aoki, Y, et al.
Udgivet: (2011)

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.
af: Aoki, Y, et al.
Udgivet: (2012)