Rationale: The Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In
Cardiac Disease (CUPID 1) study was a phase 1/phase 2 first-in-human clinical gene …

Objectives: Impaired cardiac isoform of sarco (endo) plasmic reticulum Ca2+ ATPase
(SERCA2a) activity is a key abnormality in heart failure patients with reduced ejection …

We previously showed that medium chain acyl-coenzyme A dehydrogenase (MCAD, key
regulator of fatty acid oxidation) is positively modulated in the heart by the cardioprotective …

Neuronal nitric oxide synthase (nNOS) membrane delocalization contributes to the
pathogenesis of Duchenne muscular dystrophy (DMD) by promoting functional muscle …

Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are
currently in clinical trials, but have not been thoroughly investigated for their efficacy on …

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …

Introduction: Duchenne muscular dystrophy (DMD) is a relatively common inherited disorder
caused by defective expression of the protein dystrophin. The most direct approach to …

Restoring dystrophin expression in the muscles of patients with Duchenne muscular
dystrophy (DMD) may halt or reverse the degenerative wasting and weakness that causes …

Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle-wasting disease caused by
mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of …

Background—Heart failure is characterized by impaired function and disturbed Ca2+
homeostasis. Transgenic increases in inhibitor-1 activity have been shown to improve Ca2 …