Work

The Role of Immunosuppression in Gene- and Cell-Based Treatments for Duchenne Muscular Dystrophy

Year: 2007

Type: article

Abstract: Duchenne muscular dystrophy (DMD) is a progressive and lethal, X-linked muscle-wasting disease caused by mutations in the dystrophin gene, affecting 1 in 3,500 males born worldwide. Clinically, most boys with DMD exhibit muscle weakness by the age of 5 years and lose independent ambulation by 10–12 years. During the late teen years, respiratory failure occurs and pulmonary ventilatory support is often required. Although this strategy allows patients to survive into their third decade, the onset of cardiomyopathy and worsening of respiratory function prevent longer-term survival.

Source: Molecular Therapy

DOI: 10.1038/sj.mt.6300194

Author: Valder R Arruda

Institutions: Children's Hospital of Philadelphia, University of Pennsylvania

Language: English

Topic: Muscle Physiology and Disorders

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500