Cargando…

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphor...

Descripción completa

Detalles Bibliográficos
Autores principales:	Tsoumpra, Maria K., Fukumoto, Seiji, Matsumoto, Toshio, Takeda, Shin'ichi, Wood, Matthew J.A., Aoki, Yoshitsugu
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2019
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6642283/ https://www.ncbi.nlm.nih.gov/pubmed/31257147 http://dx.doi.org/10.1016/j.ebiom.2019.06.036

Ejemplares similares

Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy
por: Aoki, Yoshitsugu, et al.
Publicado: (2013)

Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide
por: Watanabe, Naoki, et al.
Publicado: (2023)

Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors
por: Miyatake, Shouta, et al.
Publicado: (2016)

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches
por: Shimizu-Motohashi, Yuko, et al.
Publicado: (2019)

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
por: Miskew Nichols, Bailey, et al.
Publicado: (2016)

Exon-Skipping in Duchenne Muscular Dystrophy
por: Takeda, Shin’ichi, et al.
Publicado: (2021)

Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy
por: Wilton-Clark, Harry, et al.
Publicado: (2023)

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
por: Watanabe, Naoki, et al.
Publicado: (2018)

Mammalian Models of Duchenne Muscular Dystrophy: Pathological Characteristics and Therapeutic Applications
por: Nakamura, Akinori, et al.
Publicado: (2011)

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2007)

Trends in steroid therapy for Duchenne muscular dystrophy in Japan
por: Takeuchi, Fumi, et al.
Publicado: (2016)

The Effect of 6-Thioguanine on Alternative Splicing and Antisense-Mediated Exon Skipping Treatment for Duchenne Muscular Dystrophy
por: Verhaart, Ingrid E. C., et al.
Publicado: (2012)

Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy
por: Marchesi, Elena, et al.
Publicado: (2022)

Development of outcome measures according to dystrophic phenotypes in canine X-linked muscular dystrophy in Japan
por: Kuraoka, Mutsuki, et al.
Publicado: (2021)

Comparison of Experimental Protocols of Physical Exercise for mdx Mice and Duchenne Muscular Dystrophy Patients
por: Hyzewicz, Janek, et al.
Publicado: (2015)

Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy
por: Okada, Takashi, et al.
Publicado: (2013)

Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy
por: van Westering, Tirsa L. E., et al.
Publicado: (2020)

Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
por: van Deutekom, Judith, et al.
Publicado: (2023)

Sugammadex and Reversal of Neuromuscular Block in Adult Patient with Duchenne Muscular Dystrophy
por: Wefki Abdelgawwad Shousha, Ahmed Abdelgawwad, et al.
Publicado: (2014)

Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients
por: Takeuchi, Fumi, et al.
Publicado: (2013)

A phase I study of TAS‐205 in patients with Duchenne muscular dystrophy
por: Takeshita, Eri, et al.
Publicado: (2018)

Modelling Duchenne muscular dystrophy in MYOD1-converted urine-derived cells treated with 3-deazaneplanocin A hydrochloride
por: Takizawa, Hotake, et al.
Publicado: (2019)

Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy)
por: Nakamura, Harumasa, et al.
Publicado: (2013)

The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
por: Scaglioni, Dominic, et al.
Publicado: (2021)

Identification of Muscle-Specific MicroRNAs in Serum of Muscular Dystrophy Animal Models: Promising Novel Blood-Based Markers for Muscular Dystrophy
por: Mizuno, Hideya, et al.
Publicado: (2011)

Publisher Correction: Modelling Duchenne muscular dystrophy in MYOD1-converted urine-derived cells treated with 3-deazaneplanocin A hydrochloride
por: Takizawa, Hotake, et al.
Publicado: (2020)

Mitochondria mediate cell membrane repair and contribute to Duchenne muscular dystrophy
por: Vila, Maria C, et al.
Publicado: (2017)

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle
por: Miyatake, Shouta, et al.
Publicado: (2019)

Survival in Duchenne muscular dystrophy
por: RALL, SUSANNE, et al.
Publicado: (2012)

Theragnosis for Duchenne Muscular Dystrophy
por: Luce, Leonela, et al.
Publicado: (2021)

Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
por: Jirka, Silvana M G, et al.
Publicado: (2015)

Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model
por: Relizani, Karima, et al.
Publicado: (2017)

Long-term ventilation of patients with Duchenne muscular dystrophy: experiences at the Neuromuscular Centre Ulm
por: WOLLINSKY, KURT H., et al.
Publicado: (2012)

Recent insights into neuromuscular junction biology in Duchenne muscular dystrophy: Impacts, challenges, and opportunities
por: Ng, Sean Y, et al.
Publicado: (2020)

Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections
por: Paredes-Redondo, Amaia, et al.
Publicado: (2021)

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides
por: Tone, Yuichiro, et al.
Publicado: (2021)

Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy
por: Shabanpoor, Fazel, et al.
Publicado: (2015)

Alleviation of masticatory disturbance with an occlusal splint in a Duchenne muscular dystrophy patient
por: Fukumoto, Yutaka, et al.
Publicado: (2021)

Initial Pulmonary Respiration Causes Massive Diaphragm Damage and Hyper-CKemia in Duchenne Muscular Dystrophy Dog
por: Nakamura, Akinori, et al.
Publicado: (2013)

Early phase 2 trial of TAS‐205 in patients with Duchenne muscular dystrophy
por: Komaki, Hirofumi, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_kmre1sr548ghj6scmcb6jcvpav