Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Бусад хувилбаруудыг харуулах (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...

Бүрэн тодорхойлолт

Номзүйн дэлгэрэнгүй
Үндсэн зохиолчид:	Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J
Формат:	Journal article
Хэл сонгох:	English
Хэвлэсэн:	2011

Ижил төстэй зүйлс

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
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DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
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BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model
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Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
-н: Rafael, J, зэрэг
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Targeting of CAT and VCAM1 as Novel Therapeutic Targets for DMD Cardiomyopathy
-н: Bin Li, зэрэг
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Analysis of the transcriptional control of utrophin promoters as a therapeutic strategy for Duchenne muscular dystrophy (DMD).
-н: Perkins, K, зэрэг
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-н: Maria Julia Marques
Хэвлэсэн: (2017-02-01)

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-н: Cockburn, D, зэрэг
Хэвлэсэн: (1991)

Second-generation compound for the modulation of utrophin in the therapy of DMD
-н: Guiraud, S, зэрэг
Хэвлэсэн: (2015)

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

Prime editing strategies to mediate exon skipping in DMD gene
-н: Cedric Happi Mbakam, зэрэг
Хэвлэсэн: (2023-05-01)

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
-н: Javier Poyatos-García, зэрэг
Хэвлэсэн: (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.
-н: Koenig, M, зэрэг
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Novel 5 '-utrophin isoforms exhibit species-specific transcriptional profiles and provide additional candidates for therapeutic up-regulation in DMD
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P740: DMD or not DMD? Clinical genome sequencing in the interpretation of complex copy number gains
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Duchenne muscular dystrophy (DMD) cardiomyocyte-secreted exosomes promote the pathogenesis of DMD-associated cardiomyopathy
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Хэвлэсэн: (2020-11-01)

A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20
-н: Sara Mata López, зэрэг
Хэвлэсэн: (2018-05-01)

Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model
-н: Mathilde Doisy, зэрэг
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Special Issue: Pathophysiology and Therapeutic Perspectives in DMD: The Well-Defined Role of the Immune System
-н: Andrea Farini
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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
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The analysis of DMD gene deletions by multiplex PCR in Indonesian DMD/BMD patients: the era of personalized medicine
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EXPLORING TRANSIENT, NEUTRONIC, REDUCED-ORDER MODELS USING DMD/POD-GALERKIN AND DATA-DRIVEN DMD
-н: Elzohery Rabab, зэрэг
Хэвлэсэн: (2021-01-01)

A dinucleotide repeat polymorphism at the DMD locus.
-н: Powell, J, зэрэг
Хэвлэсэн: (1991)

Generation and characterization of transgenic mice with the full-length human DMD gene.
-н: 't Hoen, P, зэрэг
Хэвлэсэн: (2008)

Identification of serum protein biomarkers for utrophin based DMD therapy.
-н: Guiraud, S, зэрэг
Хэвлэсэн: (2017)

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

Matrilineal analysis of mutations in the DMD gene in a multigenerational South Indian cohort using DMD gene panel sequencing
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Muscular dystrophy associated with the DMD gene in women
-н: E. O. Vorontsova, зэрэг
Хэвлэсэн: (2024-09-01)

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

Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
-н: Michael R. Hicks, зэрэг
Хэвлэсэн: (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
-н: Pei, Ting-Han, зэрэг
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Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype
-н: Brioschi Simona, зэрэг
Хэвлэсэн: (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
-н: Ying Bai, зэрэг
Хэвлэсэн: (2022-05-01)

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

Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD
-н: Goyenvalle, A, зэрэг
Хэвлэсэн: (2012)

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD
-н: Guiraud, S, зэрэг
Хэвлэсэн: (2018)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
-н: Godfrey, C, зэрэг
Хэвлэсэн: (2012)

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