Muscular Dystrophy

Muscular dystrophy refers to a range of muscle diseases caused by defects in muscle proteins, leading to death of the muscle cells, with loss of muscle tissue, and weakness. Here are the latest discoveries pertaining to this disease.

September 16, 2021

Bone health in Duchenne muscular dystrophy: clinical and biochemical correlates.

Journal of Endocrinological Investigation
Antonino CatalanoSonia Messina
September 16, 2021
Open Access

Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening.

Scientific Reports
Patricia Soblechero-MartínVirginia Arechavala-Gomeza
September 15, 2021

Biofabrication of 3D Human Muscle Model with Vascularization and Endomysium.

Methods in Molecular Biology
Simone BersiniMatteo Moretti
September 15, 2021
Open Access

Filopodia powered by class X myosin promote fusion of mammalian myoblasts.

ELife
David W HammersH Lee Sweeney
September 15, 2021
Open Access

Reduced mitochondrial fission and impaired energy metabolism in human primary skeletal muscle cells of Megaconial Congenital Muscular Dystrophy.

Scientific Reports
Evrim Aksu-MengesBurcu Balci-Hayta
September 15, 2021
Open Access

Translational control of polyamine metabolism by CNBP is required for Drosophila locomotor function.

ELife
Sonia ConiLaura Ciapponi
September 14, 2021

Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections.

Science Advances
Amaia Paredes-RedondoYung-Yao Lin
September 13, 2021
Preprint
Open Access

Human iPSC-Based Model Reveals NOX4 as Therapeutic Target in Duchenne Cardiomyopathy

BioRxiv : the Preprint Server for Biology
R. DuelenMaurilio Sampaolesi
September 13, 2021
Preprint
Open Access

Development of a novel startle response task in Duchenne muscular dystrophy

MedRxiv : the Preprint Server for Health Sciences
K. E. MareshFrancesco Muntoni
September 14, 2021

Clinical and genetic spectrum of a large cohort of patients with δ-sarcoglycan muscular dystrophy.

Brain : a Journal of Neurology
Jorge Alonso-PérezJordi Díaz-Manera
September 14, 2021
Open Access

Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1.

Molecular Therapy. Methods & Clinical Development
Zarife SahenkJerry R Mendell
September 14, 2021
Open Access

Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy.

Molecular Therapy. Nucleic Acids
Estefanía Cerro-HerrerosRubén Artero
September 14, 2021

Gene Therapy for Duchenne Muscular Dystrophy.

Journal of Neuromuscular Diseases
Nertiyan Elangkovan, George Dickson
September 14, 2021

Adult North Star Network (ANSN): Consensus Guideline For The Standard Of Care Of Adults With Duchenne Muscular Dystrophy.

Journal of Neuromuscular Diseases
R QuinlivanANSN
September 13, 2021

Full-length Dystrophin Restoration via Targeted Genomic Integration by AAV-CRISPR in a Humanized Mouse Model of Duchenne Muscular Dystrophy.

Molecular Therapy : the Journal of the American Society of Gene Therapy
Adrian Pickar-OliverCharles A Gersbach
September 13, 2021

FKRP mutations cause congenital muscular dystrophy 1C and limb-girdle muscular dystrophy 2I in Asian patients.

Journal of Clinical Neuroscience : Official Journal of the Neurosurgical Society of Australasia
Hiroyuki AwanoIchizo Nishino
September 11, 2021

Double Eyelid Tape as a Treatment Option for Severe Ptosis in Patients with Myotonic Dystrophy Type 1: A Case Report.

American Journal of Physical Medicine & Rehabilitation
Sangah JeongSook Joung Lee
September 11, 2021

A posthuman decentring of person-centred care.

Health Sociology Review : the Journal of the Health Section of the Australian Sociological Association
Barbara E GibsonNicola M Kayes

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