[HTML][HTML] Genetic modifiers of muscular dystrophy: implications for therapy
A Heydemann, KR Doherty, EM McNally - Biochimica et Biophysica Acta …, 2007 - Elsevier
… muscle degeneration in general. This review will discuss different genetic forms of muscular
dystrophy with an emphasis on phenotypic variability and the presence of genetic modifiers …
dystrophy with an emphasis on phenotypic variability and the presence of genetic modifiers …
Advances in genetic therapeutic strategies for Duchenne muscular dystrophy
S Guiraud, H Chen, DT Burns… - Experimental …, 2015 - Wiley Online Library
… genetically based therapies targeting the primary defect of Duchenne muscular dystrophy. …
This review provides an update on the current genetically based therapies and clinical trials …
This review provides an update on the current genetically based therapies and clinical trials …
Predicting the severity of Duchenne muscular dystrophy: implications for treatment
SF Nelson, RC Griggs - Neurology, 2011 - AAN Enterprises
… An example of such a genetic modifier identified from a genome-wide screen is the … new
therapeutic targets and provide novel insights into disease pathogenesis, even though the effect …
therapeutic targets and provide novel insights into disease pathogenesis, even though the effect …
Is it time for genetic modifiers to predict prognosis in Duchenne muscular dystrophy?
L Bello, EP Hoffman, E Pegoraro - Nature Reviews Neurology, 2023 - nature.com
… reviews genetic modifier studies in DMD to date and discusses the effect of genetic modifiers
on … (inclusion of genotype-stratified subgroup analyses) and therapeutic approaches. The …
on … (inclusion of genotype-stratified subgroup analyses) and therapeutic approaches. The …
[HTML][HTML] Genetic modifiers and phenotype of Duchenne muscular dystrophy: a systematic review and meta-analysis
C Pascual-Morena, I Cavero-Redondo, A Saz-Lara… - Pharmaceuticals, 2021 - mdpi.com
… muscular dystrophy (DMD) phenotype. This meta-analysis aims to estimate the association
of genetic … ) and secreted phosphoprotein 1 (SPP1) genes, among others, with age of loss of …
of genetic … ) and secreted phosphoprotein 1 (SPP1) genes, among others, with age of loss of …
How genetic modifiers influence the phenotype of spinal muscular atrophy and suggest future therapeutic approaches
B Wirth, L Garbes, M Riessland - Current opinion in genetics & …, 2013 - Elsevier
… a large variety of factors is spinal muscular atrophy (SMA), selected herein to exemplify the
role of modifiers and the use of this knowledge gained in developing target-driven therapies. …
role of modifiers and the use of this knowledge gained in developing target-driven therapies. …
[HTML][HTML] Causes of clinical variability in Duchenne and Becker muscular dystrophies and implications for exon skipping therapies
EP Hoffman - Acta Myologica, 2020 - ncbi.nlm.nih.gov
… genetic modifiers in DMD suggests that the ‘effect size’ of the … Effect size of genetic modifiers
can be quantitated in DMD by … Genetic modifiers to date all show about 1-2 years change in …
can be quantitated in DMD by … Genetic modifiers to date all show about 1-2 years change in …
[HTML][HTML] Genetic modifiers of Duchenne muscular dystrophy and dilated cardiomyopathy
A Barp, L Bello, L Politano, P Melacini, C Calore… - PloS one, 2015 - journals.plos.org
… We report a putative protective effect of DMD genetic modifiers on the development of
cardiac complications, that might aid in risk stratification if confirmed in independent cohorts. …
cardiac complications, that might aid in risk stratification if confirmed in independent cohorts. …
Duchenne muscular dystrophy: an updated review of common available therapies
… Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive …
on the recent gene therapies for DMD that aim to compensate for dystrophin deficiency and the …
on the recent gene therapies for DMD that aim to compensate for dystrophin deficiency and the …
Genetic modifiers of D uchenne and facioscapulohumeral muscular dystrophies
RM Hightower, MS Alexander - Muscle & nerve, 2018 - Wiley Online Library
… (mdx) mice, have yielded valuable genetic modifiers of disease pathology.32 This Review
… consequences of genetic modifiers in the 2 most prevalent forms of muscular dystrophy in …
… consequences of genetic modifiers in the 2 most prevalent forms of muscular dystrophy in …