Rescue of dystrophic skeletal muscle by PGC-1α involves restored expression of dystrophin-associated protein complex components and satellite cell signaling

K Hollinger, D Gardan-Salmon… - American Journal …, 2013 - journals.physiology.org
Duchenne muscular dystrophy is typically diagnosed in the preschool years because of
locomotor defects, indicative of muscle damage. Thus, effective therapies must be able to …

Genome editing-mediated utrophin upregulation in Duchenne muscular dystrophy stem cells

K Sengupta, MK Mishra, E Loro, MJ Spencer… - … Therapy-Nucleic Acids, 2020 - cell.com
Utrophin upregulation is considered a promising therapeutic strategy for Duchenne
muscular dystrophy (DMD). A number of microRNAs (miRNAs) post-transcriptionally …

Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials

JN Kornegay, CF Spurney, PP Nghiem… - ILAR …, 2014 - academic.oup.com
Duchenne muscular dystrophy (DMD) is an X-linked human disorder in which absence of
the protein dystrophin causes degeneration of skeletal and cardiac muscle. For the sake of …

[HTML][HTML] Pre-clinical drug tests in the mdx mouse as a model of dystrophinopathies: an overview

A De Luca - Acta myologica, 2012 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy is a lethal X-linked muscle disease affecting 1/3500 live male
birth. It results from defects in the subsarcolemmal protein dystrophin, a component of the …

Non-glycanated biglycan and LTBP4: leveraging the extracellular matrix for Duchenne muscular dystrophy therapeutics

JR Fallon, EM McNally - Matrix Biology, 2018 - Elsevier
The extracellular matrix (ECM) plays key roles in normal and diseased skeletal and cardiac
muscle. In healthy muscle the ECM is essential for transmitting contractile force, maintaining …

Genetic modifiers of D uchenne and facioscapulohumeral muscular dystrophies

RM Hightower, MS Alexander - Muscle & nerve, 2018 - Wiley Online Library
Muscular dystrophy is defined as the progressive wasting of skeletal muscles that is caused
by inherited or spontaneous genetic mutations. Next‐generation sequencing has greatly …

Micro-utrophin improves cardiac and skeletal muscle function of severely affected D2/mdx mice

TL Kennedy, S Guiraud, B Edwards, S Squire… - … Therapy-Methods & …, 2018 - cell.com
Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by
mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and …

NBD delivery improves the disease phenotype of the golden retriever model of Duchenne muscular dystrophy

JN Kornegay, JM Peterson, DJ Bogan, W Kline… - Skeletal muscle, 2014 - Springer
Background Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin
gene and afflicts skeletal and cardiac muscles. Previous studies showed that DMD is …

Role of proteoglycans in the regulation of the skeletal muscle fibrotic response

E Brandan, J Gutierrez - The FEBS journal, 2013 - Wiley Online Library
Myogenesis consists of a highly organized and regulated sequence of cellular processes
aimed at forming or repairing muscle tissue. Several processes occur during myogenesis …

Sarcospan: a small protein with large potential for Duchenne muscular dystrophy

JL Marshall, RH Crosbie-Watson - Skeletal muscle, 2013 - Springer
Purification of the proteins associated with dystrophin, the gene product responsible for
Duchenne muscular dystrophy, led to the discovery of the dystrophin-glycoprotein complex …