Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

Εμφάνιση άλλων εκδόσεων (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...

Πλήρης περιγραφή

Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς:	Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J
Μορφή:	Journal article
Γλώσσα:	English
Έκδοση:	2011

Παρόμοια τεκμήρια

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
ανά: Pichavant, C, κ.ά.
Έκδοση: (2011)

Utrophin upregulation in DMD therapy: current status and new tools for the future
ανά: Fairclough, R, κ.ά.
Έκδοση: (2011)

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
ανά: Remko Goossens, κ.ά.
Έκδοση: (2023-12-01)

BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model
ανά: SongTing Shi, κ.ά.
Έκδοση: (2011-02-01)

DMD and West syndrome
ανά: Cardas, R, κ.ά.
Έκδοση: (2017)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
ανά: Rafael, J, κ.ά.
Έκδοση: (2000)

Targeting of CAT and VCAM1 as Novel Therapeutic Targets for DMD Cardiomyopathy
ανά: Bin Li, κ.ά.
Έκδοση: (2021-04-01)

Analysis of the transcriptional control of utrophin promoters as a therapeutic strategy for Duchenne muscular dystrophy (DMD).
ανά: Perkins, K, κ.ά.
Έκδοση: (2000)

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

Analysis of DMD translocations
ανά: Cockburn, D, κ.ά.
Έκδοση: (1991)

Second-generation compound for the modulation of utrophin in the therapy of DMD
ανά: Guiraud, S, κ.ά.
Έκδοση: (2015)

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

Prime editing strategies to mediate exon skipping in DMD gene
ανά: Cedric Happi Mbakam, κ.ά.
Έκδοση: (2023-05-01)

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
ανά: Javier Poyatos-García, κ.ά.
Έκδοση: (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.
ανά: Koenig, M, κ.ά.
Έκδοση: (1987)

Novel 5 '-utrophin isoforms exhibit species-specific transcriptional profiles and provide additional candidates for therapeutic up-regulation in DMD
ανά: Perkins, K, κ.ά.
Έκδοση: (2012)

P740: DMD or not DMD? Clinical genome sequencing in the interpretation of complex copy number gains
ανά: Edward Higginbotham, κ.ά.
Έκδοση: (2024-01-01)

Duchenne muscular dystrophy (DMD) cardiomyocyte-secreted exosomes promote the pathogenesis of DMD-associated cardiomyopathy
ανά: Melanie Gartz, κ.ά.
Έκδοση: (2020-11-01)

A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20
ανά: Sara Mata López, κ.ά.
Έκδοση: (2018-05-01)

Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model
ανά: Mathilde Doisy, κ.ά.
Έκδοση: (2023-12-01)

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

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
ανά: Raffaella Willmann, κ.ά.
Έκδοση: (2020-02-01)

The analysis of DMD gene deletions by multiplex PCR in Indonesian DMD/BMD patients: the era of personalized medicine
ανά: Kristy Iskandar, κ.ά.
Έκδοση: (2019-10-01)

EXPLORING TRANSIENT, NEUTRONIC, REDUCED-ORDER MODELS USING DMD/POD-GALERKIN AND DATA-DRIVEN DMD
ανά: Elzohery Rabab, κ.ά.
Έκδοση: (2021-01-01)

A dinucleotide repeat polymorphism at the DMD locus.
ανά: Powell, J, κ.ά.
Έκδοση: (1991)

Generation and characterization of transgenic mice with the full-length human DMD gene.
ανά: 't Hoen, P, κ.ά.
Έκδοση: (2008)

Identification of serum protein biomarkers for utrophin based DMD therapy.
ανά: Guiraud, S, κ.ά.
Έκδοση: (2017)

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

Matrilineal analysis of mutations in the DMD gene in a multigenerational South Indian cohort using DMD gene panel sequencing
ανά: Arun Shastry, κ.ά.
Έκδοση: (2021-05-01)

Muscular dystrophy associated with the DMD gene in women
ανά: E. O. Vorontsova, κ.ά.
Έκδοση: (2024-09-01)

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

Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
ανά: Michael R. Hicks, κ.ά.
Έκδοση: (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
ανά: Pei, Ting-Han, κ.ά.
Έκδοση: (2024)

Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype
ανά: Brioschi Simona, κ.ά.
Έκδοση: (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
ανά: Ying Bai, κ.ά.
Έκδοση: (2022-05-01)

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

Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD
ανά: Goyenvalle, A, κ.ά.
Έκδοση: (2012)

Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD
ανά: Guiraud, S, κ.ά.
Έκδοση: (2018)

Advancing the potential of peptide-PMO compounds in exon skipping therapy for DMD
ανά: Godfrey, C, κ.ά.
Έκδοση: (2012)

A female patient carrying a novel DMD mutation with non-random X-chromosome inactivation from a DMD family
ανά: Ming-Xia Sun, κ.ά.
Έκδοση: (2024-02-01)