Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

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Duchenne muscular dystrophy (DMD) is a genetic disease affecting about one in every 3,500 boys. This X-linked pathology is due to the absence of dystrophin in muscle fibers. This lack of dystrophin leads to the progressive muscle degeneration that is often responsible for the death of the DMD patien...

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Bibliografische gegevens
Hoofdauteurs:	Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J
Formaat:	Journal article
Taal:	English
Gepubliceerd in:	2011

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Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
door: Pichavant, C, et al.
Gepubliceerd in: (2011)

Utrophin upregulation in DMD therapy: current status and new tools for the future
door: Fairclough, R, et al.
Gepubliceerd in: (2011)

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
door: Remko Goossens, et al.
Gepubliceerd in: (2023-12-01)

BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model
door: SongTing Shi, et al.
Gepubliceerd in: (2011-02-01)

DMD and West syndrome
door: Cardas, R, et al.
Gepubliceerd in: (2017)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
door: Rafael, J, et al.
Gepubliceerd in: (2000)

Targeting of CAT and VCAM1 as Novel Therapeutic Targets for DMD Cardiomyopathy
door: Bin Li, et al.
Gepubliceerd in: (2021-04-01)

Analysis of the transcriptional control of utrophin promoters as a therapeutic strategy for Duchenne muscular dystrophy (DMD).
door: Perkins, K, et al.
Gepubliceerd in: (2000)

A Potential New Therapeutic Approach to DMD: PKC Theta Inhibition
door: Maria Julia Marques
Gepubliceerd in: (2017-02-01)

Analysis of DMD translocations
door: Cockburn, D, et al.
Gepubliceerd in: (1991)

Second-generation compound for the modulation of utrophin in the therapy of DMD
door: Guiraud, S, et al.
Gepubliceerd in: (2015)

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
door: Monika Hiller, et al.
Gepubliceerd in: (2018-01-01)

Prime editing strategies to mediate exon skipping in DMD gene
door: Cedric Happi Mbakam, et al.
Gepubliceerd in: (2023-05-01)

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
door: Javier Poyatos-García, et al.
Gepubliceerd in: (2024-10-01)

Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals.
door: Koenig, M, et al.
Gepubliceerd in: (1987)

Novel 5 '-utrophin isoforms exhibit species-specific transcriptional profiles and provide additional candidates for therapeutic up-regulation in DMD
door: Perkins, K, et al.
Gepubliceerd in: (2012)

P740: DMD or not DMD? Clinical genome sequencing in the interpretation of complex copy number gains
door: Edward Higginbotham, et al.
Gepubliceerd in: (2024-01-01)

Duchenne muscular dystrophy (DMD) cardiomyocyte-secreted exosomes promote the pathogenesis of DMD-associated cardiomyopathy
door: Melanie Gartz, et al.
Gepubliceerd in: (2020-11-01)

A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20
door: Sara Mata López, et al.
Gepubliceerd in: (2018-05-01)

Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model
door: Mathilde Doisy, et al.
Gepubliceerd in: (2023-12-01)

Special Issue: Pathophysiology and Therapeutic Perspectives in DMD: The Well-Defined Role of the Immune System
door: Andrea Farini
Gepubliceerd in: (2021-12-01)

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
door: Raffaella Willmann, et al.
Gepubliceerd in: (2020-02-01)

The analysis of DMD gene deletions by multiplex PCR in Indonesian DMD/BMD patients: the era of personalized medicine
door: Kristy Iskandar, et al.
Gepubliceerd in: (2019-10-01)

EXPLORING TRANSIENT, NEUTRONIC, REDUCED-ORDER MODELS USING DMD/POD-GALERKIN AND DATA-DRIVEN DMD
door: Elzohery Rabab, et al.
Gepubliceerd in: (2021-01-01)

A dinucleotide repeat polymorphism at the DMD locus.
door: Powell, J, et al.
Gepubliceerd in: (1991)

Generation and characterization of transgenic mice with the full-length human DMD gene.
door: 't Hoen, P, et al.
Gepubliceerd in: (2008)

Identification of serum protein biomarkers for utrophin based DMD therapy.
door: Guiraud, S, et al.
Gepubliceerd in: (2017)

Guidelines for Corticosteroid use in Treatment of DMD
door: Vamshi K. Rao
Gepubliceerd in: (2016-07-01)

Matrilineal analysis of mutations in the DMD gene in a multigenerational South Indian cohort using DMD gene panel sequencing
door: Arun Shastry, et al.
Gepubliceerd in: (2021-05-01)

Muscular dystrophy associated with the DMD gene in women
door: E. O. Vorontsova, et al.
Gepubliceerd in: (2024-09-01)

Measuring of customer satisfaction on example of Delta DMD
door: Kostić Ela
Gepubliceerd in: (2010-01-01)

Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
door: Michael R. Hicks, et al.
Gepubliceerd in: (2023-08-01)

Bessel_DMD: the numerical code based on the scalar Fresnel–Kirchhoff integration to calculate the diffraction and bessel-like beam by using the DMD
door: Pei, Ting-Han, et al.
Gepubliceerd in: (2024)

Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype
door: Brioschi Simona, et al.
Gepubliceerd in: (2012-08-01)

Long-Read Sequencing Revealed Extragenic and Intragenic Duplications of Exons 56–61 in DMD in an Asymptomatic Male and a DMD Patient
door: Ying Bai, et al.
Gepubliceerd in: (2022-05-01)

Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene
door: Cedric Happi Mbakam, et al.
Gepubliceerd in: (2022-12-01)

Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD
door: Goyenvalle, A, et al.
Gepubliceerd in: (2012)

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD
door: Guiraud, S, et al.
Gepubliceerd in: (2018)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
door: Godfrey, C, et al.
Gepubliceerd in: (2012)

A female patient carrying a novel DMD mutation with non-random X-chromosome inactivation from a DMD family
door: Ming-Xia Sun, et al.
Gepubliceerd in: (2024-02-01)