Stem-Cell Research and Clinical Application Stem cells have been the focus of hope, hype,
and a great deal of research. But what exactly is a stem cell, and what are its uses? This …

Skeletal muscle is the largest organ of human body with several important roles in everyday
movement and metabolic homeostasis. The limited ability of small animal models of muscle …

Abstract Introduction/Aims With current and anticipated disease‐modifying treatments,
including gene therapy, an early diagnosis for Duchenne muscular dystrophy (DMD) is …

Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive
muscle weakness, is caused by the absence or a decreased amount of the muscle …

Heritable cardiomyopathies are a class of heart diseases caused by variations in a number
of genetic loci. Genetic variants on one allele lead to either a degraded protein, which …

Musculoskeletal injuries and disorders are the leading cause of physical disability
worldwide and a considerable socioeconomic burden. The lack of effective therapies has …

Highlights•Current therapies for autoimmunity halt natural immune surveillance.•High doses
of Factor VIII administered in hemophilia A induce tolerance.•Gene replacement therapy can …

Introduction: Progressive skeletal muscle wasting is the manifesting symptom of Duchenne
muscular dystrophy, an X-linked inherited disorder triggered by primary abnormalities in the …

Duchenne muscular dystrophy is a devastating disease that leads to progressive muscle
loss and premature death. While medical management focuses mostly on symptomatic …

Hydrodynamic limb vein injection is an in vivo locoregional gene delivery method. It consists
of administrating a large volume of solution containing nucleic acid constructs in a limb with …