Pathophysiology of cardiac hypertrophy and heart failure: signaling pathways and novel therapeutic targets

YK Tham, BC Bernardo, JYY Ooi, KL Weeks… - Archives of …, 2015 - Springer
The onset of heart failure is typically preceded by cardiac hypertrophy, a response of the
heart to increased workload, a cardiac insult such as a heart attack or genetic mutation …

The war against heart failure: the Lancet lecture

E Braunwald - The Lancet, 2015 - thelancet.com
Heart failure is a global problem with an estimated prevalence of 38 million patients
worldwide, a number that is increasing with the ageing of the population. It is the most …

Mesenchymal stem cell-based therapy for cardiovascular disease: progress and challenges

L Bagno, KE Hatzistergos, W Balkan, JM Hare - Molecular Therapy, 2018 - cell.com
Administration of mesenchymal stem cells (MSCs) to diseased hearts improves cardiac
function and reduces scar. These effects occur via stimulation of endogenous repair …

Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo …

B Greenberg, J Butler, GM Felker, P Ponikowski… - The Lancet, 2016 - thelancet.com
Summary Background Sarcoplasmic/endoplasmic reticulum Ca 2+-ATPase (SERCA2a)
activity is deficient in the failing heart. Correction of this abnormality by gene transfer might …

Small and large animal models in cardiac contraction research: advantages and disadvantages

N Milani-Nejad, PML Janssen - Pharmacology & therapeutics, 2014 - Elsevier
The mammalian heart is responsible for not only pumping blood throughout the body but
also adjusting this pumping activity quickly depending upon sudden changes in the …

Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID) a phase 2 trial of intracoronary gene therapy of sarcoplasmic …

M Jessup, B Greenberg, D Mancini, T Cappola… - Circulation, 2011 - Am Heart Assoc
Background—Adeno-associated virus type 1/sarcoplasmic reticulum Ca2+-ATPase was
assessed in a randomized, double-blind, placebo-controlled, phase 2 study in patients with …

Modulation of cardiac contractility by the phopholamban/SERCA2a regulatome

EG Kranias, RJ Hajjar - Circulation research, 2012 - Am Heart Assoc
Heart disease remains the leading cause of death and disability in the Western world.
Current therapies aim at treating the symptoms rather than the subcellular mechanisms …

Long-term effects of AAV1/SERCA2a gene transfer in patients with severe heart failure: analysis of recurrent cardiovascular events and mortality

K Zsebo, A Yaroshinsky, JJ Rudy, K Wagner… - Circulation …, 2014 - Am Heart Assoc
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 …

Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial

BE Jaski, ML Jessup, DM Mancini, TP Cappola… - Journal of cardiac …, 2009 - Elsevier
BACKGROUND: SERCA2a deficiency is commonly seen in advanced heart failure (HF).
This study is designed to investigate safety and biological effects of enzyme replacement …

Basic biology of adeno-associated virus (AAV) vectors used in gene therapy

B Balakrishnan, GR Jayandharan - Current gene therapy, 2014 - ingentaconnect.com
Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …