Cargando…

Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

Recently, several promising treatments have emerged for neuromuscular disorders, highlighting the need for robust biomarkers for monitoring therapeutic efficacy and maintenance of the therapeutic effect. Several studies have proposed circulating and tissue biomarkers, but none of them has been valid...

Descripción completa

Detalles Bibliográficos
Autores principales:	Mariot, Virginie, Le Guiner, Caroline, Barthélémy, Inès, Montus, Marie, Blot, Stéphane, Torelli, Silvia, Morgan, Jennifer, Muntoni, Francesco, Voit, Thomas, Dumonceaux, Julie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7363622/ https://www.ncbi.nlm.nih.gov/pubmed/32695843 http://dx.doi.org/10.1016/j.omtm.2020.06.016

Ejemplares similares

Gene Editing to Tackle Facioscapulohumeral Muscular Dystrophy
por: Mariot, Virginie, et al.
Publicado: (2022)

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
por: Le Guiner, Caroline, et al.
Publicado: (2017)

Effects of an Immunosuppressive Treatment in the GRMD Dog Model of Duchenne Muscular Dystrophy
por: Barthélémy, Inès, et al.
Publicado: (2012)

A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy
por: Mariot, Virginie, et al.
Publicado: (2020)

Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy
por: Barthélémy, Inès, et al.
Publicado: (2014)

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

Replenishing NAD(+) content reduces aspects of striated muscle disease in a dog model of Duchenne muscular dystrophy
por: Cardoso, Déborah, et al.
Publicado: (2023)

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
por: Zhou, Haiyan, et al.
Publicado: (2020)

Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
por: Mariot, Virginie, et al.
Publicado: (2017)

Quantifying the burden of caregiving in Duchenne muscular dystrophy
por: Landfeldt, Erik, et al.
Publicado: (2016)

Spatial and Temporal Non-Uniform Changes in Left Ventricular Myocardial Strain in Dogs with Duchenne Muscular Dystrophy
por: Ghaleh, Bijan, et al.
Publicado: (2023)

A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples
por: Sardone, Valentina, et al.
Publicado: (2018)

Gait characterization in golden retriever muscular dystrophy dogs using linear discriminant analysis
por: Fraysse, Bodvaël, et al.
Publicado: (2017)

miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy
por: Sanson, M., et al.
Publicado: (2020)

Two novel missense mutations in the myostatin gene identified in Japanese patients with Duchenne muscular dystrophy
por: Nishiyama, Atsushi, et al.
Publicado: (2007)

The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle
por: Rybalka, Emma, et al.
Publicado: (2020)

Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy
por: Larcher, Thibaut, et al.
Publicado: (2014)

RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
por: Domenger, Claire, et al.
Publicado: (2017)

Growth pattern trajectories in boys with Duchenne muscular dystrophy
por: Stimpson, Georgia, et al.
Publicado: (2022)

Levels of inflammation and oxidative stress, and a role for taurine in dystropathology of the Golden Retriever Muscular Dystrophy dog model for Duchenne Muscular Dystrophy
por: Terrill, Jessica R., et al.
Publicado: (2016)

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

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
por: Frank, Diane E., et al.
Publicado: (2020)

Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead
por: Heslop, Emma, et al.
Publicado: (2021)

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

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

Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy
por: Chesshyre, Mary, et al.
Publicado: (2022)

Development of a novel startle response task in Duchenne muscular dystrophy
por: Maresh, Kate, et al.
Publicado: (2022)

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy
por: Zaharieva, Irina T., et al.
Publicado: (2013)

Comparative proteomic analyses of Duchenne muscular dystrophy and Becker muscular dystrophy muscles: changes contributing to preserve muscle function in Becker muscular dystrophy patients
por: Capitanio, Daniele, et al.
Publicado: (2020)

Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy
por: Zambon, Alberto A., et al.
Publicado: (2022)

Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy
por: Amor, Fatima, et al.
Publicado: (2021)

Hypokalemia complicating Duchenne muscular dystrophy.
por: McDonald, B., et al.
Publicado: (1987)

The role of fibrosis in Duchenne muscular dystrophy
por: KLINGLER, WERNER, et al.
Publicado: (2012)

Delay in Diagnosis of Duchenne Muscular Dystrophy
por: Rao, Vamshi K., et al.
Publicado: (2015)

Circulating Biomarkers for Duchenne Muscular Dystrophy
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2015)

Targeting angiogenesis in Duchenne muscular dystrophy
por: Podkalicka, Paulina, et al.
Publicado: (2019)

Regenerative biomarkers for Duchenne muscular dystrophy
por: Guiraud, Simon, et al.
Publicado: (2019)

Eteplirsen in the treatment of Duchenne muscular dystrophy
por: Lim, Kenji Rowel Q, et al.
Publicado: (2017)

Nutritional Challenges in Duchenne Muscular Dystrophy
por: Salera, Simona, et al.
Publicado: (2017)

Lifetime Care of Duchenne Muscular Dystrophy
por: MacKintosh, Erin W., et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_bfg6okt514ogi2aeqvnclb7evi