Year: 2017
Type: article
Abstract: Recent clinical gene therapy trials for the treatment of heart failure have failed to meet primary efficacy endpoints and have tampered enthusiasm for the future application of cardiac gene therapy. These results have brought to light the difficulty of efficiently introducing genes into the human heart and have focused on potential problems that need to be addressed before further clinical applications. These trials however have established the safety of gene delivery vectors for cardiac targeting in humans. The sinusoidal trajectory of gene therapy continues and despite these setbacks the future of the field is promising.
Source: Circulation Research
Authors: Roger J. Hajjar, Kiyotake Ishikawa
Institution: Icahn School of Medicine at Mount Sinai
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: bronze
Awards: C-TRIP: Targeted Gene Therapy for the Treatment of Heart Failure (P20), Targeting Abnormal Calcium Cycling Using Novel Gene Therapy Vectors, Calcium Pump Activators for Heart Failure Therapy, TRIP: Targeted Gene Therapy for the Treatment of Heart Failure, G5456674798, Role of miR25 in Heart Failure, Training Program in Molecular and Cellular Cardiology, SUMO1 and SERCA2a Function, Treating Ventricle and Valve: New Synergies for Ischemic LV Remodeling with MR, Anti-AAV Antibodies as an Obstacle to Cardiac AAV Gene Therapy
OpenAlex ID: W2569554011