Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?

T Yokota, W Duddy, Y Echigoya… - Expert opinion on …, 2012 - Taylor & Francis
Introduction: Duchenne muscular dystrophy (DMD), one of the most common and lethal
genetic disorders, is caused by mutations of the dystrophin gene. Removal of an exon or of …

[PDF][PDF] Dystrophin levels required for generic correction of Duchenne muscular dystrophy

JS Chamberlain - BAM-PADOVA-, 1997 - bio.unipd.it
Correction of the muscle pathology in Duchenne muscular dystrophy (DMD) could
theoretically be achieved if methods are developed to produce functional versions of the …

Cloning of the Duchenne/Becker muscular dystrophy locus

AP Monaco, LM Kunkel - Advances in Human Genetics 1, 1988 - Springer
The identification and molecular cloning of the Duchenne muscular dystrophy (DMD) gene
represents the first step in the long path toward a basic understanding and potential therapy …

Harnessing the potential of dystrophin‐related proteins for ameliorating Duchenne's muscular dystrophy

TOB Krag, M Gyrd‐Hansen… - Acta physiologica …, 2001 - Wiley Online Library
Duchenne's muscular dystrophy (DMD) is a fatal disease caused by mutations in the DMD
gene that lead to quantitative and qualitative disturbances in dystrophin expression …

Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy

A Govoni, F Magri, S Brajkovic, C Zanetta… - Cellular and molecular …, 2013 - Springer
Muscular dystrophy is a heterogeneous group of genetic disorders characterised by
progressive muscle tissue degeneration. No effective treatment has been discovered for …

Novel therapies for Duchenne muscular dystrophy

R Kapsa, AJ Kornberg, E Byrne - The Lancet Neurology, 2003 - thelancet.com
The development of therapeutic strategies that overcome the unique problems posed by
Duchenne muscular dystrophy (DMD) has lead to the development of many contemporary …

Pharmacological strategies for muscular dystrophy

TS Khurana, KE Davies - Nature Reviews Drug Discovery, 2003 - nature.com
Duchenne muscular dystrophy (DMD) is a fatal, genetic disorder whose relentless
progression underscores the urgency for developing a cure. Although Duchenne initiated …

[HTML][HTML] Antisense and gene therapy options for Duchenne muscular dystrophy arising from mutations in the N-terminal hotspot

H Wilton-Clark, T Yokota - Genes, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is a fatal genetic disease affecting children that is
caused by a mutation in the gene encoding for dystrophin. In the absence of functional …

Common therapeutic advances for Duchenne muscular dystrophy (DMD)

A Salmaninejad, Y Jafari Abarghan… - International Journal …, 2021 - Taylor & Francis
Background and purpose: Duchenne muscular dystrophy (DMD), a lethal X-linked recessive
muscle dystrophy, is resulted in by different mutations including mostly frame-shifting gross …

[HTML][HTML] Teaching an old molecule new tricks: Drug repositioning for Duchenne muscular dystrophy

L Vitiello, L Tibaudo, E Pegoraro, L Bello… - International Journal of …, 2019 - mdpi.com
Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular
dystrophies. The disease is caused by the lack of dystrophin, a structurally essential protein; …