Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Show other versions (1)

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...

תיאור מלא

מידע ביבליוגרפי
Main Authors:	Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J
פורמט:	Journal article
שפה:	English
יצא לאור:	2011

פריטים דומים

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
מאת: Pichavant, C, et al.
יצא לאור: (2011)

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

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

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

DMD and West syndrome
מאת: Cardas, R, et al.
יצא לאור: (2017)

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

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

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

A Potential New Therapeutic Approach to DMD: PKC Theta Inhibition
מאת: Maria Julia Marques
יצא לאור: (2017-02-01)

Analysis of DMD translocations
מאת: Cockburn, D, et al.
יצא לאור: (1991)

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

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

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

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
מאת: Javier Poyatos-García, et al.
יצא לאור: (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.
מאת: Koenig, M, et al.
יצא לאור: (1987)

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

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

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

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

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

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

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

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

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

A dinucleotide repeat polymorphism at the DMD locus.
מאת: Powell, J, et al.
יצא לאור: (1991)

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

Identification of serum protein biomarkers for utrophin based DMD therapy.
מאת: Guiraud, S, et al.
יצא לאור: (2017)

Guidelines for Corticosteroid use in Treatment of DMD
מאת: Vamshi K. Rao
יצא לאור: (2016-07-01)

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

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

Measuring of customer satisfaction on example of Delta DMD
מאת: Kostić Ela
יצא לאור: (2010-01-01)

Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
מאת: Michael R. Hicks, et al.
יצא לאור: (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
מאת: Pei, Ting-Han, et al.
יצא לאור: (2024)

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

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

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

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

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

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