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

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

Splice-switching antisense oligonucleotides are emerging treatments for neuromuscular diseases, with several splice-switching oligonucleotides (SSOs) currently undergoing clinical trials such as for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). However, the development of syst...

Full description

Bibliographic Details
Main Authors:	Shabanpoor, F, Hammond, S, Abendroth, F, Hazell, G, Wood, M, Gait, M
Format:	Journal article
Language:	English
Published:	Mary Ann Liebert 2017

Similar Items

Evaluation of cell-penetrating peptide delivery of antisense oligonucleotides for therapeutic efficacy in spinal muscular atrophy
by: Hammond, S, et al.
Published: (2019)

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
by: Hammond, S, et al.
Published: (2016)

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy
by: Suzan M. Hammond, et al.
Published: (2022-12-01)

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model
by: Juri Hasegawa, et al.
Published: (2024-09-01)

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

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
by: Wood, M, et al.
Published: (2017)

Rapid screening of gene function by systemic delivery of morpholino oligonucleotides to live mouse embryos.
by: Kathryn S McClelland, et al.
Published: (2015-01-01)

A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy
by: Jeffrey M Keil, et al.
Published: (2014-01-01)

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy
by: Meijboom, KE, et al.
Published: (2021)

Aminoglycoside Enhances the Delivery of Antisense Morpholino Oligonucleotides In Vitro and in mdx Mice
by: Mingxing Wang, et al.
Published: (2019-06-01)

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

Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
by: James S. Novak, et al.
Published: (2017-10-01)

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy
by: Katharina E. Meijboom, et al.
Published: (2021-07-01)

LONG-TERM RELEASE DELIVERY METHOD OF ANTISENSE OLIGONUCLEOTIDES IN HIPSC DERIVED MOTONEURONS FROM SPINAL MUSCULAR ATROPHY DISEASE
by: Maria Victoria Martinez Dominguez, et al.
Published: (2023-10-01)

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy
by: Gogliotti, R, et al.
Published: (2010)

Cell penetrating peptide delivery of splice directing oligonucleotides as a treatment for Duchenne muscular dystrophy.
by: Betts, C, et al.
Published: (2013)

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
by: Haiyan Zhou, et al.
Published: (2020-06-01)

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA
by: Peter Järver, et al.
Published: (2012-01-01)

Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
by: James S. Novak, et al.
Published: (2018-03-01)

Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
by: James S. Novak, et al.
Published: (2018-01-01)

Vivo-Morpholinos: A non-peptide transporter delivers Morpholinos into a wide array of mouse tissues
by: Paul A. Morcos, et al.
Published: (2008-12-01)

Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene
by: Eric W Ottesen, et al.
Published: (2024-02-01)

Assessment of Nuclear Gem Quantity for Evaluating the Efficacy of Antisense Oligonucleotides in Spinal Muscular Atrophy Cells
by: Haya Al-Hilal, et al.
Published: (2024-01-01)

Splice-switching therapy for spinal muscular atrophy.
by: Meijboom, K, et al.
Published: (2017)

Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients
by: Lars Hendrik Müschen, et al.
Published: (2021-02-01)

Spinal muscular atrophy.
by: Talbot, K, et al.
Published: (2001)

Spinal Muscular Atrophy
by: J Gordon Millichap
Published: (1990-02-01)

Spinal muscular atrophy
by: D'Amico Adele, et al.
Published: (2011-11-01)

Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides.
by: Te-Lin Lin, et al.
Published: (2016-01-01)

PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy.
by: Hammond, S, et al.
Published: (2010)

Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
by: Wang M, et al.
Published: (2015-09-01)

Synthesis of Backbone-Modified Morpholino Oligonucleotides Using Phosphoramidite Chemistry
by: Sibasish Paul, et al.
Published: (2023-07-01)

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy.
by: Sleigh, J, et al.
Published: (2011)

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy
by: James N. Sleigh, et al.
Published: (2011-07-01)

A Semi‐Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy
by: Konstantinos Biliouris, et al.
Published: (2018-09-01)

Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.
by: Palittiya Sintusek, et al.
Published: (2016-01-01)

Efficient delivery of DNA and morpholinos into mouse preimplantation embryos by electroporation.
by: Hui Peng, et al.
Published: (2012-01-01)

Spinal Muscular Atrophy and Arthrogryposis
by: J Gordon Millichap
Published: (1997-10-01)

Rehabilitation in spinal muscular atrophy
by: Agus Iwan Foead, et al.
Published: (2019-01-01)

Rehabilitation in spinal muscular atrophy
by: Joanna Iłżecka
Published: (2021-05-01)