Cargando…

Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression

High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials. Safety concerns with this therapeutic approach include the immune response to introdu...

Descripción completa

Detalles Bibliográficos
Autores principales:	Starikova, Anna V., Skopenkova, Victoria V., Polikarpova, Anna V., Reshetov, Denis A., Vassilieva, Svetlana G., Velyaev, Oleg A., Shmidt, Anna A., Savchenko, Irina M., Soldatov, Vladislav O., Egorova, Tatiana V., Bardina, Maryana V.
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/PMC8764061/ https://www.ncbi.nlm.nih.gov/pubmed/35039573 http://dx.doi.org/10.1038/s41598-022-04892-x

Ejemplares similares

CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein
por: Egorova, Tatiana V., et al.
Publicado: (2023)

CRISPR/Cas9-generated mouse model with humanizing single-base substitution in the Gnao1 for safety studies of RNA therapeutics
por: Polikarpova, Anna V., et al.
Publicado: (2023)

In-Frame Deletion of Dystrophin Exons 8–50 Results in DMD Phenotype
por: Egorova, Tatiana V., et al.
Publicado: (2023)

Muscle-Specific Promoters for Gene Therapy
por: Skopenkova, V. V., et al.
Publicado: (2021)

CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene
por: Egorova, Tatiana V., et al.
Publicado: (2019)

Adeno-Associated Viruses for Modeling Neurological Diseases in Animals: Achievements and Prospects
por: Lunev, Evgenii, et al.
Publicado: (2022)

PCR-Based Analytical Methods for Quantification and Quality Control of Recombinant Adeno-Associated Viral Vector Preparations
por: Shmidt, Anna A., et al.
Publicado: (2021)

Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects
por: Banks, Glen B., et al.
Publicado: (2020)

Characterization of the HIV-1 RNA associated proteome identifies Matrin3 and PSF as nuclear cofactors of Rev function
por: Kula, Anna, et al.
Publicado: (2011)

Combined Heterogeneous Catalyst Based on Titanium Oxide for Highly Efficient Oxidative Desulfurization of Model Fuels
por: Polikarpova, Polina D., et al.
Publicado: (2022)

Recognition of 3′ nucleotide context and stop codon readthrough are determined during mRNA translation elongation
por: Biziaev, Nikita, et al.
Publicado: (2022)

Post transcriptional regulation of HIV-1 gene expression by MATR3, PSF and Rev
por: Gharu, Lavina, et al.
Publicado: (2013)

Current advances in gene therapy of mitochondrial diseases
por: Soldatov, Vladislav O., et al.
Publicado: (2022)

Correction: Current advances in gene therapy of mitochondrial diseases
por: Soldatov, Vladislav O., et al.
Publicado: (2023)

Polarization of Acoustic Waves in Two-Dimensional Phononic Crystals Based on Fused Silica
por: Marunin, Mikhail V., et al.
Publicado: (2022)

Old-Developed Areas and Socio-Geographic Space of Russia: From the Editors of the Special Issue
por: Nefedova, T. G., et al.
Publicado: (2023)

Machine Learning Assisted Classification of Cell Lines and Cell States on Quantitative Phase Images
por: Belashov, Andrey V., et al.
Publicado: (2021)

Synergistic Anti-Angiogenic Effects Using Peptide-Based Combinatorial Delivery of siRNAs Targeting VEGFA, VEGFR1, and Endoglin Genes
por: Egorova, Anna A., et al.
Publicado: (2019)

N (3)-[(E)-Morpholin-4-ylmethylidene]-1-phenyl-1H-1,2,4-triazole-3,5-diamine monohydrate
por: Chernyshev, V. M., et al.
Publicado: (2010)

3,5-Diamino-1-phenyl-1,2,4-triazolium bromide
por: Chernyshev, V. M., et al.
Publicado: (2010)

CodonLogo: a sequence logo-based viewer for codon patterns
por: Sharma, Virag, et al.
Publicado: (2012)

A Novel C1q Domain-Containing Protein Isolated from the Mollusk Modiolus kurilensis Recognizing Glycans Enriched with Acidic Galactans and Mannans
por: Grinchenko, Andrei V., et al.
Publicado: (2021)

Anti-IL-17 monoclonal antibodies in hospitalized patients with severe COVID-19: A pilot study
por: Avdeev, Sergey N., et al.
Publicado: (2021)

Molecular Factors Involved in Spinal Muscular Atrophy Pathways as Possible Disease-modifying Candidates
por: Maretina, Marianna A., et al.
Publicado: (2018)

2-(4-Chlorobenzoyl)-1-(diaminomethylene)hydrazinium chloride monohydrate
por: Chernyshev, V. M., et al.
Publicado: (2010)

Base-Calling Algorithm with Vocabulary (BCV) Method for Analyzing Population Sequencing Chromatograms
por: Fantin, Yuri S., et al.
Publicado: (2013)

ZLIB++: object-oriented numerical library for differential algebra
por: Malitsky, N, et al.
Publicado: (1994)

Translation at first sight: the influence of leading codons
por: Osterman, Ilya A, et al.
Publicado: (2020)

B Cell Regulation in Autoimmune Diseases
por: Sokolov, A. V., et al.
Publicado: (2018)

A Cross-Sectional Study of the Gut Microbiota Composition in Moscow Long-Livers
por: Kashtanova, Daria A., et al.
Publicado: (2020)

N-Docosahexaenoylethanolamine Attenuates Neuroinflammation and Improves Hippocampal Neurogenesis in Rats with Sciatic Nerve Chronic Constriction Injury
por: Tyrtyshnaia, Anna A., et al.
Publicado: (2020)

Application of Microsatellite Loci for Molecular Identification of Elite Genotypes, Analysis of Clonality, and Genetic Diversity in Aspen Populus tremula L. (Salicaceae)
por: Politov, Dmitry V., et al.
Publicado: (2015)

Retrospective Study of Listeria monocytogenes Isolated in the Territory of Inner Eurasia from 1947 to 1999
por: Psareva, Ekaterina K., et al.
Publicado: (2019)

Object-oriented approach for the design of the simulation facility of the SSC
por: Bourianoff, G I, et al.
Publicado: (1994)

PAC++: object-oriented platform for accelerator codes
por: Malitsky, N, et al.
Publicado: (1994)

Synthesis, Characterization and Biodistribution of GdF(3):Tb(3+)@RB Nanocomposites
por: Polozhentsev, Oleg E., et al.
Publicado: (2022)

Low toxic maghemite nanoparticles for theranostic applications
por: Kuchma, Elena A, et al.
Publicado: (2017)

Codons Support the Maintenance of Intrinsic DNA Polymer Flexibility over Evolutionary Timescales
por: Babbitt, G. A., et al.
Publicado: (2012)

Investigating the Coating Effect on Charge Transfer Mechanisms in Composite Electrodes for Lithium-Ion Batteries
por: Fedorova, Anna A., et al.
Publicado: (2023)

Neuroprotective action of Cortexin, Cerebrolysin and Actovegin in acute or chronic brain ischemia in rats
por: Kurkin, Denis V., et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_s66pnvc9f6t56hbdu2dsmjikv9