BACKGROUND: Heart failure (HF) remains a major cause of morbidity and mortality in North
America. With an aging population and an unmet clinical need by current pharmacologic …

Aims δ-Sarcoglycan is a member of the dystrophin-associated glycoprotein complex linking
the cytoskeleton to the extracellular matrix. Similar to patients with defects in the gene …

As a prerequisite for clinical application, we determined the long-term therapeutic
effectiveness and safety of adeno-associated virus (AAV)–S100A1 gene therapy in a …

Adenoviral gene transfer of sarco (endo) plasmic reticulum Ca2+-ATPase (SERCA) 2a to the
hypertrophic heart in vivo has been consistently reported to lead to enhanced myocardial …

Background—The incidence of heart failure is ever-growing, and it is urgent to develop
improved treatments. An attractive approach is gene therapy; however, the clinical barrier …

Abstract Adeno-associated virus (AAV)-mediated microdystrophin gene therapy holds great
promise for treating Duchenne muscular dystrophy (DMD). Previous studies have revealed …

Systemic delivery of genes to muscle using vectors based on recombinant
adenovirusassociated virus (rAAV) has been explored extensively in animal models of …

Aims Restoration of sarco/endoplasmic reticulum Ca2+ ATPase (SERCA2a) activity through
gene transfer improved cardiac function in experimental and pilot studies in humans with …

Background—Sarcoplasmic reticulum calcium ATPase 2a (SERCA2a) gene therapy
improves mechanical function in heart failure and is under evaluation in a clinical trial. A …

Trans-splicing adeno-associated viral (tsAAV) vectors hold great promise for delivering large
therapeutic genes. One potential application is in the treatment of Duchenne muscular …