Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Adeno-associated virus (AAV)-mediated gene therapy for Duchenne muscular dystrophy: the issue of transgene persistence

A Manini, E Abati, A Nuredini, S Corti… - Frontiers in …, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …

AAV-based gene therapies for the muscular dystrophies

JM Crudele, JS Chamberlain - Human molecular genetics, 2019 - academic.oup.com
Muscular dystrophy (MD) is a group of progressive genetic diseases affecting the
musculature that are characterized by inflammatory infiltrates, necrosis and connective …

The mdx mouse model as a surrogate for D uchenne muscular dystrophy

TA Partridge - The FEBS journal, 2013 - Wiley Online Library
Research into fundamental principles and the testing of therapeutic hypotheses for treatment
of human disease is commonly performed on mouse models of human diseases. Although …

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

A Filareto, S Parker, R Darabi, L Borges… - Nature …, 2013 - nature.com
Duchenne muscular dystrophy is a progressive and incurable neuromuscular disease
caused by genetic and biochemical defects of the dystrophin–glycoprotein complex. Here …

The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies

GB Banks, JS Chamberlain - Current topics in developmental biology, 2008 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy.
There is no effective treatment and patients typically die in approximately the third decade …

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping

A Goyenvalle, A Babbs, D Powell, R Kole, S Fletcher… - Molecular Therapy, 2010 - cell.com
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by
mutations in the dystrophin gene that result in the absence of functional protein. Antisense …

Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models

Y Song, L Morales, AS Malik, AF Mead, CD Greer… - Nature medicine, 2019 - nature.com
The essential product of the Duchenne muscular dystrophy (DMD) gene is dystrophin, a rod-
like protein that protects striated myocytes from contraction-induced injury,. Dystrophin …

[HTML][HTML] Pharmacological TRPC6 inhibition improves survival and muscle function in mice with Duchenne muscular dystrophy

BL Lin, JY Shin, WPD Jeffreys, N Wang, CA Lukban… - JCI insight, 2022 - ncbi.nlm.nih.gov
Gene mutations causing loss of dystrophin result in the severe muscle disease known as
Duchenne muscular dystrophy (DMD). Despite efforts at genetic repair, DMD therapy …