Cargando…

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive disorder caused by mutations in the DMD gene and the subsequent lack of dystrophin protein. Recently, phosphorodiamidate morpholino oligomer (PMO)-antisense oligonucleotides (ASOs) targeting exon 51 or 53 to reestablish the DMD reading...

Descripción completa

Detalles Bibliográficos
Autores principales:	Echigoya, Yusuke, Trieu, Nhu, Duddy, William, Moulton, Hong M., Yin, HaiFang, Partridge, Terence A., Hoffman, Eric P., Kornegay, Joe N., Rohret, Frank A., Rogers, Christopher S., Yokota, Toshifumi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8657897/ https://www.ncbi.nlm.nih.gov/pubmed/34884867 http://dx.doi.org/10.3390/ijms222313065

Ejemplares similares

In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy
por: Echigoya, Yusuke, et al.
Publicado: (2015)

Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges
por: Echigoya, Yusuke, et al.
Publicado: (2018)

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts
por: Lee, Joshua, et al.
Publicado: (2018)

Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy
por: Kemaladewi, Dwi U, et al.
Publicado: (2011)

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice
por: Echigoya, Yusuke, et al.
Publicado: (2015)

Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy
por: Lim, Kenji Rowel Q., et al.
Publicado: (2022)

Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish
por: Berger, Joachim, et al.
Publicado: (2011)

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

Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy
por: Nguyen, Quynh, et al.
Publicado: (2017)

Elusive sources of variability of dystrophin rescue by exon skipping
por: Vila, Maria Candida, et al.
Publicado: (2015)

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
por: Greer, Kane L, et al.
Publicado: (2014)

CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy
por: Wilton-Clark, Harry, et al.
Publicado: (2023)

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
por: Yin, HaiFang, et al.
Publicado: (2013)

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
por: Yin, HaiFang, et al.
Publicado: (2020)

Development of an orally available inhibitor of CLK1 for skipping a mutated dystrophin exon in Duchenne muscular dystrophy
por: Sako, Yukiya, et al.
Publicado: (2017)

Effective Exon Skipping and Dystrophin Restoration by 2′-O-Methoxyethyl Antisense Oligonucleotide in Dystrophin-Deficient Mice
por: Yang, Lu, et al.
Publicado: (2013)

Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy
por: Novak, James S., et al.
Publicado: (2021)

Multiple exon skipping strategies to by-pass dystrophin mutations
por: Adkin, Carl F., et al.
Publicado: (2012)

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice
por: Han, Gang, et al.
Publicado: (2016)

Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene
por: Greer, Kane, et al.
Publicado: (2020)

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

Current Outline of Exon Skipping Trials in Duchenne Muscular Dystrophy
por: Eser, Gökçe, et al.
Publicado: (2022)

MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy
por: Barbash, Israel M., et al.
Publicado: (2012)

Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts
por: Lorain, Stéphanie, et al.
Publicado: (2010)

Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy
por: Cao, Limin, et al.
Publicado: (2014)

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
por: Fletcher, Sue, et al.
Publicado: (2012)

Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy
por: Lee, Joshua J.A., et al.
Publicado: (2018)

Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy
por: Cao, Limin, et al.
Publicado: (2018)

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies
por: Toh, Zhi Yon Charles, et al.
Publicado: (2016)

Skipping Multiple Exons to Treat DMD—Promises and Challenges
por: Aslesh, Tejal, et al.
Publicado: (2018)

Chemical treatment enhances skipping of a mutated exon in the dystrophin gene
por: Nishida, Atsushi, et al.
Publicado: (2011)

A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis
por: Anwar, Saeed, et al.
Publicado: (2021)

Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy
por: van Vliet, Laura, et al.
Publicado: (2008)

Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis
por: Shimizu-Motohashi, Yuko, et al.
Publicado: (2018)

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

Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors
por: Gapinske, Michael, et al.
Publicado: (2023)

A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping
por: Walmsley, Gemma L., et al.
Publicado: (2010)

Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries
por: Adams, Abbie M, et al.
Publicado: (2007)

Causes of clinical variability in Duchenne and Becker muscular dystrophies and implications for exon skipping therapies
por: Hoffman, Eric P.
Publicado: (2020)

Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23
por: Aartsma-Rus, Annemieke
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_c69nf3a4ghokm43ir1na9ajcgn