At the crossroads of clinical and preclinical research for muscular dystrophy—are we closer to effective treatment for patients?
KI Gawlik - International journal of molecular sciences, 2018 - mdpi.com
Among diseases affecting skeletal muscle, muscular dystrophy is one of the most
devastating and complex disorders. The term 'muscular dystrophy'refers to a heterogeneous …
devastating and complex disorders. The term 'muscular dystrophy'refers to a heterogeneous …
Biomimetic strategies for the deputization of proteoglycan functions
Proteoglycans (PGs), which have glycosaminoglycan chains attached to their protein cores,
are essential for maintaining the morphology and function of healthy body tissues …
are essential for maintaining the morphology and function of healthy body tissues …
Comparative genomics of X-linked muscular dystrophies: the golden retriever model
C Brinkmeyer-Langford, JN Kornegay - Current genomics, 2013 - ingentaconnect.com
Duchenne muscular dystrophy (DMD) is a devastating disease that dramatically decreases
the lifespan and abilities of affected young people. The primary molecular cause of the …
the lifespan and abilities of affected young people. The primary molecular cause of the …
Converging pathways involving microRNA-206 and the RNA-binding protein KSRP control post-transcriptionally utrophin A expression in skeletal muscle
A Amirouche, H Tadesse, P Miura… - Nucleic acids …, 2014 - academic.oup.com
Several reports have previously highlighted the potential role of miR-206 in the post-
transcriptional downregulation of utrophin A in cultured cells. Along those lines, we recently …
transcriptional downregulation of utrophin A in cultured cells. Along those lines, we recently …
Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy
RJ Fairclough, KJ Perkins, KE Davies - Current gene therapy, 2012 - ingentaconnect.com
DMD is a devastatingly progressive muscle wasting disorder of childhood that significantly
shortens life expectancy. Despite efforts to develop an effective therapy that dates back over …
shortens life expectancy. Despite efforts to develop an effective therapy that dates back over …
PDE10A inhibition reduces the manifestation of pathology in DMD zebrafish and represses the genetic modifier PITPNA
MR Lambert, JM Spinazzola, JJ Widrick, A Pakula… - Molecular Therapy, 2021 - cell.com
Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in
the DMD gene. Absence of dystrophin protein leads to progressive degradation of skeletal …
the DMD gene. Absence of dystrophin protein leads to progressive degradation of skeletal …
Protein-Anchoring Therapy of Biglycan for Mdx Mouse Model of Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating muscle disease caused by loss-of-
function mutations in DMD encoding dystrophin. No rational therapy is currently available …
function mutations in DMD encoding dystrophin. No rational therapy is currently available …
[PDF][PDF] ECM-related myopathies and muscular dystrophies: pros and cons of protein therapies
PM Van Ry, TM Fontelonga, P Barraza-Flores… - Compr …, 2017 - researchgate.net
Extracellular matrix (ECM) myopathies and muscular dystrophies are a group of genetic
diseases caused by mutations in genes encoding proteins that provide critical links between …
diseases caused by mutations in genes encoding proteins that provide critical links between …
Gene expression profiling identifies molecular pathways associated with collagen VI deficiency and provides novel therapeutic targets
S Paco, SG Kalko, C Jou, MA Rodríguez, J Corbera… - PLoS …, 2013 - journals.plos.org
Ullrich congenital muscular dystrophy (UCMD), caused by collagen VI deficiency, is a
common congenital muscular dystrophy. At present, the role of collagen VI in muscle and the …
common congenital muscular dystrophy. At present, the role of collagen VI in muscle and the …
Dystrophic cardiomyopathy—potential role of calcium in pathogenesis, treatment and novel therapies
Duchenne muscular dystrophy (DMD) is caused by defects in the DMD gene and results in
progressive wasting of skeletal and cardiac muscle due to an absence of functional …
progressive wasting of skeletal and cardiac muscle due to an absence of functional …