Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Atrophy occurs in specific muscles with inactivity (for example, during plaster cast
immobilization) or denervation (for example, in patients with spinal cord injuries). Muscle …

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Skeletal muscle is composed of a large and heterogeneous assortment of cell populations
that interact with each other to maintain muscle homeostasis and orchestrate regeneration …

Muscle wasting is a consequence of physiological changes or a pathology characterized by
increased catabolic activity that leads to progressive loss of skeletal muscle mass and …

Spinal muscular atrophy (SMA), the most common autosomal recessive neurodegenerative
disease affecting children, results in impaired motor neuron function. Despite knowledge of …

The discovery of naturally occurring adeno-associated virus (AAV) isolates in different
animal species and the generation of engineered AAV strains using molecular genetics …

Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD
gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life …