Cargando…

Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1

Myotonic dystrophy, or dystrophia myotonica type 1 (DM1), is a multi-systemic disorder and is the most common adult form of muscular dystrophy. It affects not only muscles but also many organs, including the brain. Cerebral impairments include cognitive deficits, daytime sleepiness, and loss of visu...

Descripción completa

Detalles Bibliográficos
Autores principales:	Ait Benichou, Siham, Jauvin, Dominic, De Serres-Bérard, Thiéry, Pierre, Marion, Ling, Karen K., Bennett, C. Frank, Rigo, Frank, Gourdon, Genevieve, Chahine, Mohamed, Puymirat, Jack
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9750879/ https://www.ncbi.nlm.nih.gov/pubmed/35075265 http://dx.doi.org/10.1038/s41434-022-00316-7

Ejemplares similares

Recent Progress and Challenges in the Development of Antisense Therapies for Myotonic Dystrophy Type 1
por: De Serres-Bérard, Thiéry, et al.
Publicado: (2022)

Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice
por: Jauvin, Dominic, et al.
Publicado: (2017)

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
por: Klein, Arnaud F., et al.
Publicado: (2019)

Electrophysiological basis of cardiac arrhythmia in a mouse model of myotonic dystrophy type 1
por: Ginjupalli, Vamsi Krishna Murthy, et al.
Publicado: (2023)

Antisense oligonucleotide and adjuvant exercise therapy reverse fatigue in old mice with myotonic dystrophy
por: Hu, Ningyan, et al.
Publicado: (2020)

Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
por: Gourdon, Genevieve, et al.
Publicado: (2017)

Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides
por: Ward, Amanda J., et al.
Publicado: (2014)

Of Mice and Men: Advances in the Understanding of Neuromuscular Aspects of Myotonic Dystrophy
por: Braz, Sandra O., et al.
Publicado: (2018)

Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides
por: Wang, Zhenxun, et al.
Publicado: (2012)

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1
por: Stoodley, Jessica, et al.
Publicado: (2023)

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2
por: Scoles, Daniel R., et al.
Publicado: (2017)

Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides
por: Scharner, Juergen, et al.
Publicado: (2020)

Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy
por: Bouwman, Linde F., et al.
Publicado: (2021)

RBFOX1 Cooperates with MBNL1 to Control Splicing in Muscle, Including Events Altered in Myotonic Dystrophy Type 1
por: Klinck, Roscoe, et al.
Publicado: (2014)

Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy
por: Lutz, Maggie, et al.
Publicado: (2023)

Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice
por: Sinha, Rahul, et al.
Publicado: (2018)

Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model
por: Sheng, Lei, et al.
Publicado: (2020)

Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
por: Nomakuchi, Tomoki T., et al.
Publicado: (2015)

Functional and histopathological identification of the respiratory failure in a DMSXL transgenic mouse model of myotonic dystrophy
por: Panaite, Petrica-Adrian, et al.
Publicado: (2013)

Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic Dystrophy
por: González-Barriga, Anchel, et al.
Publicado: (2013)

Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
por: Hu, Ningyan, et al.
Publicado: (2018)

iPSC-derived cardiomyocytes from patients with myotonic dystrophy type 1 have abnormal ion channel functions and slower conduction velocities
por: Poulin, Hugo, et al.
Publicado: (2021)

Cardiac involvement in patient-specific induced pluripotent stem cells of myotonic dystrophy type 1: unveiling the impact of voltage-gated sodium channels
por: Pierre, Marion, et al.
Publicado: (2023)

Myotonic dystrophy
por: Patterson, VH
Publicado: (1990)

Synaptic protein dysregulation in myotonic dystrophy type 1: Disease neuropathogenesis beyond missplicing
por: Hernández-Hernández, Oscar, et al.
Publicado: (2013)

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

Antisense oligonucleotide therapy for KCNT1 encephalopathy
por: Burbano, Lisseth Estefania, et al.
Publicado: (2022)

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

Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction
por: Wang, Lingyan, et al.
Publicado: (2020)

Cancer and Myotonic Dystrophy
por: D’Ambrosio, Eleonora S., et al.
Publicado: (2023)

Targeting nuclear RNA for in vivo correction of myotonic dystrophy
por: Wheeler, Thurman M., et al.
Publicado: (2012)

Relationships between grip strength, myotonia, and CTG expansion in myotonic dystrophy type 1
por: Hogrel, Jean‐Yves, et al.
Publicado: (2017)

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice
por: Toonen, Lodewijk J.A., et al.
Publicado: (2017)

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

Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy
por: Wojtkowiak-Szlachcic, Agnieszka, et al.
Publicado: (2015)

Antisense transcription of the myotonic dystrophy locus yields low-abundant RNAs with and without (CAG)n repeat
por: Gudde, Anke E. E. G., et al.
Publicado: (2017)

Characterization of RAN Translation and Antisense Transcription in Primary Cell Cultures of Patients with Myotonic Dystrophy Type 1
por: Koehorst, Emma, et al.
Publicado: (2021)

Efficient electroporation of neuronal cells using synthetic oligonucleotides: identifying duplex RNA and antisense oligonucleotide activators of human frataxin expression
por: Shen, Xiulong, et al.
Publicado: (2019)

Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment
por: Ponnath, Abhilash, et al.
Publicado: (2017)

Intracerebral Infusion of Antisense Oligonucleotides Into Prion-infected Mice
por: Nazor Friberg, Karah, et al.
Publicado: (2012)

Cannot write session to /tmp/vufind_sessions/sess_gcmh0o8d5ujpbkgdg9ugr6o287