Lista de obras -

Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.

artículo científico publicado en 2014

Design and application of bispecific splice-switching oligonucleotides

artículo científico publicado en 2014

Genetic therapies for RNA mis-splicing diseases ⬇️

artículo científico publicado el 15 de abril de 2011

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

artículo científico publicado en 2015

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

artículo científico publicado en 2017

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy.

artículo científico publicado en 2015

Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice.

artículo científico publicado en 2018

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy.

artículo científico publicado en 2009

Mouse survival motor neuron alleles that mimic SMN2 splicing and are inducible rescue embryonic lethality early in development but not late

artículo científico publicado en 2010

Negative regulatory elements are present in the human LMO2 oncogene and may contribute to its expression in leukemia.

artículo científico publicado en 2005

Optimizing Tissue-Specific Antisense Oligonucleotide–Peptide Conjugates ⬇️

artículo científico publicado el 1 de enero de 2012

PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy.

artículo científico publicado en 2010

Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides.

artículo científico publicado en 2015

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

artículo científico publicado en 2011

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

artículo científico publicado en 2012

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

artículo científico publicado en 2015

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

artículo científico publicado en 2016

Targeting the 5' untranslated region of <i>SMN2</i> as a therapeutic strategy for spinal muscular atrophy

artículo científico publicado en 2021

Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.

artículo científico publicado en 2015

Use of cell-penetrating-peptides in oligonucleotide splice switching therapy.

artículo científico publicado en 2012

Lista de obras de

Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.

Design and application of bispecific splice-switching oligonucleotides

Genetic therapies for RNA mis-splicing diseases ⬇️

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy.

Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice.

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy.

Mouse survival motor neuron alleles that mimic SMN2 splicing and are inducible rescue embryonic lethality early in development but not late

Negative regulatory elements are present in the human LMO2 oncogene and may contribute to its expression in leukemia.

Optimizing Tissue-Specific Antisense Oligonucleotide–Peptide Conjugates ⬇️

PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy.

Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides.

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

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

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

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Targeting the 5' untranslated region of <i>SMN2</i> as a therapeutic strategy for spinal muscular atrophy

Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.

Use of cell-penetrating-peptides in oligonucleotide splice switching therapy.