Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice. (Q35084227)

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English	Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.	scientific article

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29 January 2020

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice (English)

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29 January 2020

Patrizia Camelliti

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29 January 2020

Suzan M Hammond

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29 January 2020

Matthew J. Wood

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29 January 2020

author name string

HaiFang Yin

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29 January 2020

Amer F Saleh

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29 January 2020

Corinne Betts

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29 January 2020

Andrey Arzumanov

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29 January 2020

Thomas Merritt

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29 January 2020

Michael J Gait

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publication date

19 April 2011

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29 January 2020

Molecular Therapy

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29 January 2020

19

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29 January 2020

7

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29 January 2020

1295-1303

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29 January 2020

Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO.

1 reference

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8 July 2018

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.

1 reference

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8 July 2018

RNA-targeted splice-correction therapy for neuromuscular disease

1 reference

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8 July 2018

Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

8 July 2018

Heart-specific overexpression of CUGBP1 reproduces functional and molecular abnormalities of myotonic dystrophy type 1.

1 reference

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8 July 2018

PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1.

1 reference

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8 July 2018

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

1 reference

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8 July 2018

Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle.

1 reference

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8 July 2018

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

1 reference

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8 July 2018

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

1 reference

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8 July 2018

Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides

1 reference

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8 July 2018

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

8 July 2018

Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

8 July 2018

Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

8 July 2018

Interaction of nitric oxide synthase with the postsynaptic density protein PSD-95 and alpha1-syntrophin mediated by PDZ domains

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

8 July 2018

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

8 July 2018

Tissue slices from adult mammalian hearts as a model for pharmacological drug testing

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3128823

27 September 2018

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

1 reference

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27 September 2018

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

https://pubmed.ncbi.nlm.nih.gov/21505427

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Toward an oligonucleotide therapy for Duchenne muscular dystrophy: a complex development challenge

1 reference

https://pubmed.ncbi.nlm.nih.gov/21505427

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Serum CK, calcium, magnesium, and oxidative phosphorylation in mdx mouse muscular dystrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/21505427

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A rat antibody against a structure functionally related to the mouse T-cell receptor/T3 complex

1 reference

https://pubmed.ncbi.nlm.nih.gov/21505427

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1038/MT.2011.79

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29 January 2020

1 reference

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29 January 2020

1 reference

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29 January 2020

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