AAV6-βARKct gene delivery mediated by molecular cardiac surgery with recirculating delivery (MCARD) in sheep results in robust gene expression and increased adrenergic reserve

J Thorac Cardiovasc Surg. 2012 Mar;143(3):720-726.e3. doi: 10.1016/j.jtcvs.2011.08.048. Epub 2011 Dec 3.

Abstract

Objective: Genetic modulation of heart function is a novel therapeutic strategy. We investigated the effect of molecular cardiac surgery with recirculating delivery (MCARD)-mediated carboxyl-terminus of the β-adrenergic receptor kinase (βARKct) gene transfer on cardiac mechanoenergetics and β-adrenoreceptor (βAR) signaling.

Methods: After baseline measurements, sheep underwent MCARD-mediated delivery of 10(14) genome copies of self-complimentary adeno-associated virus (scAAV6)-βARKct. Four and 8 weeks after MCARD, mechanoenergetic studies using magnetic resonance imaging were performed. Tissues were analyzed with real-time quantitative polymerase chain reaction (RT-qPCR) and Western blotting. βAR density, cyclic adenosine monophosphate levels, and physiologic parameters were evaluated.

Results: There was a significant increase in dP/dt(max) at 4 weeks: 1384 ± 76 versus 1772 ± 182 mm Hg/s; and the increase persisted at 8 weeks in response to isoproterenol (P < .05). Similarly, the magnitude of dP/dt(min) increased at both 4 weeks and 8 weeks with isoproterenol stimulation (P < .05). At 8 weeks, potential energy was conserved, whereas in controls there was a decrease in potential energy (P < .05) in response to isoproterenol. RT-qPCR confirmed robustness of βARKct expression throughout the left ventricle and undetectable expression in extracardiac tissues. Quantitative Western blot data confirmed higher expression of βARKct in the left ventricle: 0.46 ± 0.05 versus 0.00 in lung and liver (P < .05). Survival was 100% and laboratory parameters of major organ function were within normal limits.

Conclusions: MCARD-mediated βARKct delivery is safe, results in robust cardiac-specific gene expression, enhances cardiac contractility and lusitropy, increases adrenergic reserve, and improves energy utilization efficiency in a preclinical large animal model.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Adrenergic beta-Agonists / pharmacology
  • Animals
  • Blotting, Western
  • Cardiac Surgical Procedures*
  • Cyclic AMP / metabolism
  • Dependovirus / genetics*
  • Echocardiography, Doppler
  • Gene Expression Regulation
  • Gene Transfer Techniques*
  • Genetic Vectors*
  • Heart Ventricles / diagnostic imaging
  • Heart Ventricles / drug effects
  • Heart Ventricles / enzymology*
  • Hemodynamics
  • Isoproterenol / pharmacology
  • Magnetic Resonance Imaging
  • Male
  • Myocardial Contraction
  • Real-Time Polymerase Chain Reaction
  • Receptors, Adrenergic, beta / drug effects
  • Receptors, Adrenergic, beta / genetics
  • Receptors, Adrenergic, beta / metabolism*
  • Sheep
  • Signal Transduction* / drug effects
  • Signal Transduction* / genetics
  • Time Factors
  • Ventricular Pressure
  • beta-Adrenergic Receptor Kinases / biosynthesis*
  • beta-Adrenergic Receptor Kinases / genetics

Substances

  • Adrenergic beta-Agonists
  • Receptors, Adrenergic, beta
  • Cyclic AMP
  • beta-Adrenergic Receptor Kinases
  • Isoproterenol