Cargando…

Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice

Detalles Bibliográficos
Autores principales:	Karuppasamy, Muthukumar, Alexander, Matthew S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Commentary
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10238456/ https://www.ncbi.nlm.nih.gov/pubmed/37273785 http://dx.doi.org/10.1016/j.omtn.2023.04.007

Ejemplares similares

Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping
por: Omairi, Saleh, et al.
Publicado: (2017)

Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression
por: Maino, Eleonora, et al.
Publicado: (2021)

Dystrophin immunogenicity and requirement in myogenic cells: Paradigm shift in gene therapy for DMD
por: Górecki, Dariusz C.
Publicado: (2022)

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

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method
por: Iyombe-Engembe, Jean-Paul, et al.
Publicado: (2016)

Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes
por: Xiao, Rou, et al.
Publicado: (2022)

Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmd(mdx) rat model
por: Caudal, Dorian, et al.
Publicado: (2020)

Corrigendum to “Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method”
Publicado: (2016)

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

Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X‐Linked (mdx) Mouse
por: Zarrouki, Faouzi, et al.
Publicado: (2022)

Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide-Morpholino Conjugate in Dystrophin-Deficient mdx Mice
por: Gao, Xianjun, et al.
Publicado: (2020)

Restoration of dystrophin expression using the Sleeping Beauty transposon
por: Muses, Sofia, et al.
Publicado: (2012)

Dystrophin (DMD) Missense Variant in Cats with Becker-Type Muscular Dystrophy
por: Hilton, Stephanie, et al.
Publicado: (2023)

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

Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy
por: Aupy, Philippine, et al.
Publicado: (2019)

Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice
por: Karri, Dileep R., et al.
Publicado: (2022)

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
por: Hanson, Britt, et al.
Publicado: (2022)

Screening of Dystrophin Gene Deletions in Egyptian Patients with DMD/BMD Muscular Dystrophies
por: Effat, Laila K., et al.
Publicado: (2000)

Notch signalling restores satellite stem cell activity in a model of DMD
Publicado: (2014)

Resveratrol Decreases Oxidative Stress by Restoring Mitophagy and Improves the Pathophysiology of Dystrophin-Deficient mdx Mice
por: Sebori, Rio, et al.
Publicado: (2018)

Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle
por: Morin, Adrien, et al.
Publicado: (2023)

Transplanted Human Oligodendrocyte Progenitor Cells Restore Neurobehavioral Deficits in a Rat Model of Preterm White Matter Injury
por: Wang, Xiaohua, et al.
Publicado: (2021)

Spermidine rejuvenates T lymphocytes and restores anticancer immunosurveillance in aged mice
por: Castoldi, Francesca, et al.
Publicado: (2022)

DMDtoolkit: a tool for visualizing the mutated dystrophin protein and predicting the clinical severity in DMD
por: Zhou, Jiapeng, et al.
Publicado: (2017)

Characterization of a Dmd(EGFP) reporter mouse as a tool to investigate dystrophin expression
por: Petkova, Mina V., et al.
Publicado: (2016)

The Role of Dystrophin Gene Mutations in Neuropsychological Domains of DMD Boys: A Longitudinal Study
por: Tyagi, Rahul, et al.
Publicado: (2020)

Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration
por: Roberts, Thomas C., et al.
Publicado: (2015)

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

Restorative treatments of dystrophin expression in Duchenne muscular dystrophy: A systematic review
por: Pascual‐Morena, Carlos, et al.
Publicado: (2020)

Restoring normoglycaemia: not so harmless
por: Preiser, Jean-Charles
Publicado: (2008)

CRISPR/Cas9‐Based Dystrophin Restoration Reveals a Novel Role for Dystrophin in Bioenergetics and Stress Resistance of Muscle Progenitors
por: Matre, Polina R., et al.
Publicado: (2019)

Intronic Alternative Polyadenylation in the Middle of the DMD Gene Produces Half-Size N-Terminal Dystrophin with a Potential Implication of ECG Abnormalities of DMD Patients
por: Rani, Abdul Qawee Mahyoob, et al.
Publicado: (2020)

Intraarterial Injection of Muscle-Derived Cd34(+)Sca-1(+) Stem Cells Restores Dystrophin in mdx Mice
por: Torrente, Yuan, et al.
Publicado: (2001)

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches
por: Lorain, Stéphanie, et al.
Publicado: (2013)

Sensitivity and Frequencies of Dystrophin Gene Mutations in Thai DMD/BMD Patients As Detected by Multiplex PCR
por: Sura, Thanyachai, et al.
Publicado: (2008)

Splicing Characteristics of Dystrophin Pseudoexons and Identification of a Novel Pathogenic Intronic Variant in the DMD Gene
por: Xie, Zhiying, et al.
Publicado: (2020)

Inhibitory Control Over Ca(2+) Sparks via Mechanosensitive Channels Is Disrupted in Dystrophin Deficient Muscle but Restored by Mini-Dystrophin Expression
por: Teichmann, Martin D. H., et al.
Publicado: (2008)

Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides
por: Goyenvalle, Aurelie, et al.
Publicado: (2016)

Engraftment of human induced pluripotent stem cell-derived myogenic progenitors restores dystrophin in mice with duchenne muscular dystrophy
por: He, Ruojie, et al.
Publicado: (2020)

CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice
por: Stephenson, Anthony A., et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_fl7kffqviomsvk3ah6f9m7hr6d