Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Zobrazit další vydání (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...

Podrobná bibliografie
Hlavní autoři:	Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J
Médium:	Journal article
Jazyk:	English
Vydáno:	2011

Podobné jednotky

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
Autor: Pichavant, C, a další
Vydáno: (2011)

Utrophin upregulation in DMD therapy: current status and new tools for the future
Autor: Fairclough, R, a další
Vydáno: (2011)

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
Autor: Remko Goossens, a další
Vydáno: (2023-12-01)

BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model
Autor: SongTing Shi, a další
Vydáno: (2011-02-01)

DMD and West syndrome
Autor: Cardas, R, a další
Vydáno: (2017)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
Autor: Rafael, J, a další
Vydáno: (2000)

Targeting of CAT and VCAM1 as Novel Therapeutic Targets for DMD Cardiomyopathy
Autor: Bin Li, a další
Vydáno: (2021-04-01)

Analysis of the transcriptional control of utrophin promoters as a therapeutic strategy for Duchenne muscular dystrophy (DMD).
Autor: Perkins, K, a další
Vydáno: (2000)

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

Analysis of DMD translocations
Autor: Cockburn, D, a další
Vydáno: (1991)

Second-generation compound for the modulation of utrophin in the therapy of DMD
Autor: Guiraud, S, a další
Vydáno: (2015)

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
Autor: Monika Hiller, a další
Vydáno: (2018-01-01)

Prime editing strategies to mediate exon skipping in DMD gene
Autor: Cedric Happi Mbakam, a další
Vydáno: (2023-05-01)

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
Autor: Javier Poyatos-García, a další
Vydáno: (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.
Autor: Koenig, M, a další
Vydáno: (1987)

Novel 5 '-utrophin isoforms exhibit species-specific transcriptional profiles and provide additional candidates for therapeutic up-regulation in DMD
Autor: Perkins, K, a další
Vydáno: (2012)

P740: DMD or not DMD? Clinical genome sequencing in the interpretation of complex copy number gains
Autor: Edward Higginbotham, a další
Vydáno: (2024-01-01)

Duchenne muscular dystrophy (DMD) cardiomyocyte-secreted exosomes promote the pathogenesis of DMD-associated cardiomyopathy
Autor: Melanie Gartz, a další
Vydáno: (2020-11-01)

A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20
Autor: Sara Mata López, a další
Vydáno: (2018-05-01)

Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model
Autor: Mathilde Doisy, a další
Vydáno: (2023-12-01)

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

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
Autor: Raffaella Willmann, a další
Vydáno: (2020-02-01)

The analysis of DMD gene deletions by multiplex PCR in Indonesian DMD/BMD patients: the era of personalized medicine
Autor: Kristy Iskandar, a další
Vydáno: (2019-10-01)

EXPLORING TRANSIENT, NEUTRONIC, REDUCED-ORDER MODELS USING DMD/POD-GALERKIN AND DATA-DRIVEN DMD
Autor: Elzohery Rabab, a další
Vydáno: (2021-01-01)

A dinucleotide repeat polymorphism at the DMD locus.
Autor: Powell, J, a další
Vydáno: (1991)

Generation and characterization of transgenic mice with the full-length human DMD gene.
Autor: 't Hoen, P, a další
Vydáno: (2008)

Identification of serum protein biomarkers for utrophin based DMD therapy.
Autor: Guiraud, S, a další
Vydáno: (2017)

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

Matrilineal analysis of mutations in the DMD gene in a multigenerational South Indian cohort using DMD gene panel sequencing
Autor: Arun Shastry, a další
Vydáno: (2021-05-01)

Muscular dystrophy associated with the DMD gene in women
Autor: E. O. Vorontsova, a další
Vydáno: (2024-09-01)

Measuring of customer satisfaction on example of Delta DMD
Autor: Kostić Ela
Vydáno: (2010-01-01)

Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
Autor: Michael R. Hicks, a další
Vydáno: (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
Autor: Pei, Ting-Han, a další
Vydáno: (2024)

Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype
Autor: Brioschi Simona, a další
Vydáno: (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
Autor: Ying Bai, a další
Vydáno: (2022-05-01)

Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene
Autor: Cedric Happi Mbakam, a další
Vydáno: (2022-12-01)

Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD
Autor: Goyenvalle, A, a další
Vydáno: (2012)

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD
Autor: Guiraud, S, a další
Vydáno: (2018)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
Autor: Godfrey, C, a další
Vydáno: (2012)

A female patient carrying a novel DMD mutation with non-random X-chromosome inactivation from a DMD family
Autor: Ming-Xia Sun, a další
Vydáno: (2024-02-01)