UNASSIGNED: Gene therapy is a promising new treatment option for @entity299 . For finding the most suitable and safe vector for cardiac gene transfer, we delivered adenovirus (AdV), adeno-associated virus (AAV) and lentivirus (LeV) vectors into the @entity19 heart with sophisticated closed-chest echocardiography-guided intramyocardial injection method for comparing them with regards to transduction efficiency, @entity296 , effects on the left ventricular function and electrocardiography (ECG). AdV had the highest transduction efficiency in cardiomyocytes followed by @entity4700 and AAV9, and the lowest efficiency was seen with LeV. The local @entity296 and @entity82 in the left ventricle (LV) was proportional to transduction efficiency. AdV caused @entity384 and @entity299 . Neither of the locally injected AAV serotypes impaired the LV systolic function, but AAV9 caused @entity67 to some extent. LeV did not affect the cardiac function. We also studied systemic delivery of AAV9, which led to transduction of cardiomyocytes throughout the myocardium. However, also diffuse @entity82 was present leading to significantly impaired LV systolic and diastolic function and pathological ECG changes. Compared with widely used AdV vector, @entity4700 , AAV9 and LeV were less effective in transducing cardiomyocytes but also less harmful. Local administration of AAV9 was safer and more efficient compared with systemic administration.Gene Therapy advance online publication, 28 January 2016; doi:10.1038/gt.2015.114.

Efficacy and safety of myocardial gene transfer of adenovirus, adeno-associated virus and lentivirus vectors in the XXXX heart.

@entity82 :: ('MESH:D005355', 'Disease') :: ['fibrosis'] @entity296 :: ('MESH:D009202', 'Disease') :: ['myocardial damage', 'myocardial inflammation'] @entity19 :: ('10090', 'Species') :: ['mouse'] @entity384 :: ('MESH:D002311', 'Disease') :: ['LV dilatation'] @entity299 :: ('MESH:D006331', 'Disease') :: ['cardiac diseases', 'systolic dysfunction'] @entity4700 :: ('10804', 'Species') :: ['AAV2'] @entity67 :: ('MESH:D018754', 'Disease') :: ['diastolic dysfunction']
@entity19 :: (10090,Species) :: ['mouse']
