Cell-penetrating peptide conjugates of steric blocking oligonucleotides as therapeutics for neuromuscular diseases from a historical perspective to current prospects of treatment

Cell-penetrating peptide conjugates of steric blocking oligonucleotides as therapeutics for neuromuscular diseases from a historical perspective to current prospects of treatment

The review starts with a historical perspective of the achievements of the Gait group in synthesis of oligonucleotides (ONs) and their peptide conjugates toward the award of the 2017 Oligonucleotide Therapeutic Society Lifetime Achievement Award. This acts as a prelude to the rewarding collaborative...

Mô tả đầy đủ

Chi tiết về thư mục
Những tác giả chính:	Gait, M, Arzumanov, A, McClorey, G, Godfrey, C, Betts, C, Hammond, S, Wood, M
Định dạng:	Journal article
Ngôn ngữ:	English
Được phát hành:	Mary Ann Liebert 2018

Những quyển sách tương tự

Cell-penetrating peptides to enhance delivery of Oligonucleotide-based therapeutics
Bằng: McClorey, G, et al.
Được phát hành: (2018)

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

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
Bằng: Godfrey, C, et al.
Được phát hành: (2012)

Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides
Bằng: Tone, Y, et al.
Được phát hành: (2021)

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

Optimizing tissue-specific antisense oligonucleotide-peptide conjugates.
Bằng: Betts, C, et al.
Được phát hành: (2012)

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

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

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

PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation.
Bằng: Ezzat, K, et al.
Được phát hành: (2011)

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

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

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

Design and application of bispecific splice-switching oligonucleotides.
Bằng: Bestas, B, et al.
Được phát hành: (2014)

Use of cell-penetrating-peptides in oligonucleotide splice switching therapy.
Bằng: El Andaloussi, SA, et al.
Được phát hành: (2012)

Peptide-mediated cell and in vivo delivery of antisense oligonucleotides and siRNA
Bằng: Järver, P, et al.
Được phát hành: (2012)

Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA
Bằng: Jamie Leckie, et al.
Được phát hành: (2024-06-01)

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Bằng: Klein, AF, et al.
Được phát hành: (2019)

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.
Bằng: Yin, H, et al.
Được phát hành: (2008)

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

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.
Bằng: Alison M Blain, et al.
Được phát hành: (2018-01-01)

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA
Bằng: Peter Järver, et al.
Được phát hành: (2012-01-01)

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

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy
Bằng: Shabanpoor, F, et al.
Được phát hành: (2017)

Peptide conjugation to enhance oligonucleotide delivery
Bằng: Wolfe, Justin Mahoney
Được phát hành: (2018)

Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse.
Bằng: Yin, H, et al.
Được phát hành: (2010)

Peptide–oligonucleotide conjugates as nanoscale building blocks for assembly of an artificial three-helix protein mimic
Bằng: Chenguang Lou, et al.
Được phát hành: (2016-07-01)

Peptide–Oligonucleotide Conjugation: Chemistry and Therapeutic Applications
Bằng: Anna L. Malinowska, et al.
Được phát hành: (2024-09-01)

Chemistry of Peptide-Oligonucleotide Conjugates: A Review
Bằng: Kristina Klabenkova, et al.
Được phát hành: (2021-09-01)

Emerging oligonucleotide therapeutics for rare neuromuscular diseases
Bằng: Aoki, Y, et al.
Được phát hành: (2021)

Covalently attached intercalators restore duplex stability and splice-switching activity to triazole-modified oligonucleotides
Bằng: Dysko, A, et al.
Được phát hành: (2022)

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

Enhancing the Effectiveness of Oligonucleotide Therapeutics Using Cell-Penetrating Peptide Conjugation, Chemical Modification, and Carrier-Based Delivery Strategies
Bằng: Saeed Anwar, et al.
Được phát hành: (2023-04-01)

Scavenger receptor-mediated uptake of cell-penetrating peptide nanocomplexes with oligonucleotides.
Bằng: Ezzat, K, et al.
Được phát hành: (2012)

Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides
Bằng: Wolfe, Justin, et al.
Được phát hành: (2020)

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

Profile of circadianly regulated metabolic genes in dystrophic heart
Bằng: Betts, C, et al.
Được phát hành: (2018)

Oligonucleotide-based therapies for neuromuscular disease
Bằng: Douglas, A, et al.
Được phát hành: (2015)

Coupling Strategies for the Synthesis of Peptide-Oligonucleotide Conjugates for Patterned Synthetic Biomineralization
Bằng: Joshua D. Carter, et al.
Được phát hành: (2011-01-01)

Allele-specific RNA interference to target a model of slow-channel congenital myasthenic syndrome
Bằng: McClorey, G, et al.
Được phát hành: (2008)