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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Príomhchruthaitheoirí:	Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J
Formáid:	Journal article
Teanga:	English
Foilsithe / Cruthaithe:	2011

Míreanna comhchosúla

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
de réir: Pichavant, C, et al.
Foilsithe / Cruthaithe: (2011)

Utrophin upregulation in DMD therapy: current status and new tools for the future
de réir: Fairclough, R, et al.
Foilsithe / Cruthaithe: (2011)

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
de réir: Remko Goossens, et al.
Foilsithe / Cruthaithe: (2023-12-01)

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

DMD and West syndrome
de réir: Cardas, R, et al.
Foilsithe / Cruthaithe: (2017)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
de réir: Rafael, J, et al.
Foilsithe / Cruthaithe: (2000)

Targeting of CAT and VCAM1 as Novel Therapeutic Targets for DMD Cardiomyopathy
de réir: Bin Li, et al.
Foilsithe / Cruthaithe: (2021-04-01)

Analysis of the transcriptional control of utrophin promoters as a therapeutic strategy for Duchenne muscular dystrophy (DMD).
de réir: Perkins, K, et al.
Foilsithe / Cruthaithe: (2000)

A Potential New Therapeutic Approach to DMD: PKC Theta Inhibition
de réir: Maria Julia Marques
Foilsithe / Cruthaithe: (2017-02-01)

Analysis of DMD translocations
de réir: Cockburn, D, et al.
Foilsithe / Cruthaithe: (1991)

Second-generation compound for the modulation of utrophin in the therapy of DMD
de réir: Guiraud, S, et al.
Foilsithe / Cruthaithe: (2015)

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

Prime editing strategies to mediate exon skipping in DMD gene
de réir: Cedric Happi Mbakam, et al.
Foilsithe / Cruthaithe: (2023-05-01)

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
de réir: Javier Poyatos-García, et al.
Foilsithe / Cruthaithe: (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.
de réir: Koenig, M, et al.
Foilsithe / Cruthaithe: (1987)

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

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

Duchenne muscular dystrophy (DMD) cardiomyocyte-secreted exosomes promote the pathogenesis of DMD-associated cardiomyopathy
de réir: Melanie Gartz, et al.
Foilsithe / Cruthaithe: (2020-11-01)

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

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

Special Issue: Pathophysiology and Therapeutic Perspectives in DMD: The Well-Defined Role of the Immune System
de réir: Andrea Farini
Foilsithe / Cruthaithe: (2021-12-01)

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
de réir: Raffaella Willmann, et al.
Foilsithe / Cruthaithe: (2020-02-01)

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

EXPLORING TRANSIENT, NEUTRONIC, REDUCED-ORDER MODELS USING DMD/POD-GALERKIN AND DATA-DRIVEN DMD
de réir: Elzohery Rabab, et al.
Foilsithe / Cruthaithe: (2021-01-01)

A dinucleotide repeat polymorphism at the DMD locus.
de réir: Powell, J, et al.
Foilsithe / Cruthaithe: (1991)

Generation and characterization of transgenic mice with the full-length human DMD gene.
de réir: 't Hoen, P, et al.
Foilsithe / Cruthaithe: (2008)

Identification of serum protein biomarkers for utrophin based DMD therapy.
de réir: Guiraud, S, et al.
Foilsithe / Cruthaithe: (2017)

Guidelines for Corticosteroid use in Treatment of DMD
de réir: Vamshi K. Rao
Foilsithe / Cruthaithe: (2016-07-01)

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

Muscular dystrophy associated with the DMD gene in women
de réir: E. O. Vorontsova, et al.
Foilsithe / Cruthaithe: (2024-09-01)

Measuring of customer satisfaction on example of Delta DMD
de réir: Kostić Ela
Foilsithe / Cruthaithe: (2010-01-01)

Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
de réir: Michael R. Hicks, et al.
Foilsithe / Cruthaithe: (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
de réir: Pei, Ting-Han, et al.
Foilsithe / Cruthaithe: (2024)

Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype
de réir: Brioschi Simona, et al.
Foilsithe / Cruthaithe: (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
de réir: Ying Bai, et al.
Foilsithe / Cruthaithe: (2022-05-01)

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

Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD
de réir: Goyenvalle, A, et al.
Foilsithe / Cruthaithe: (2012)

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD
de réir: Guiraud, S, et al.
Foilsithe / Cruthaithe: (2018)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
de réir: Godfrey, C, et al.
Foilsithe / Cruthaithe: (2012)

A female patient carrying a novel DMD mutation with non-random X-chromosome inactivation from a DMD family
de réir: Ming-Xia Sun, et al.
Foilsithe / Cruthaithe: (2024-02-01)