The SERCA-LVAD trial was a phase 2a trial assessing the safety and feasibility of delivering
an adeno-associated vector 1 carrying the cardiac isoform of the sarcoplasmic reticulum …

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 …

Heart failure is a major public health problem throughout the world and it is likely that its
prevalence will continue to grow over the next several decades. Despite advances in the …

Despite improvements in drug and device therapy for heart failure, hospitalization rates and
mortality have changed little in the past decade. Randomized clinical trials using gene …

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

Adeno-associated virus serotype 1 (AAV1) has many advantages as a gene therapy vector,
but the presence of pre-existing neutralizing antibodies (NAbs) is an important limitation …

Summary Background Sarcoplasmic/endoplasmic reticulum Ca 2+-ATPase (SERCA2a)
activity is deficient in the failing heart. Correction of this abnormality by gene transfer might …

Aims Down‐regulation of sarcoplasmic reticulum calcium ATPase (SERCA2a) is a key
molecular abnormality in heart failure (HF), which is not currently addressed by specific …

BACKGROUND: SERCA2a deficiency is commonly seen in advanced heart failure (HF).
This study is designed to investigate safety and biological effects of enzyme replacement …

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