Safety of gene transfer to treat heart failure supports further clinical development

June 6, 2017
Credit: Mary Ann Liebert, Inc., publishers

Based on the encouraging safety data that has emerged from multiple clinical trials that used different gene transfer approaches to improve heart function in patients suffering from heart failure with reduced ejection fraction, researchers conclude that this therapeutic strategy can be advanced with acceptable risk. They review the results of completed clinical studies and identify the current challenges in an article published in Human Gene Therapy.

In the article entitled "Randomized Clinical Trials of Gene Transfer for Heart Failure with Reduced Ejection Fraction," William Penny and H. Kirk Hammond, VA San Diego Healthcare System and University of California, San Diego, describe the different genes delivered to the patients and their intended effects, and the two gene delivery methods used in the clinical trials—injection of a virus vector into the heart muscle or delivery of a plasmid to the endocardium, the membrane lining the chambers of the heart. Despite a promising safety profile for in these patients, these early studies yielded mixed results in terms of efficacy, necessitating further development and testing.

"Complex multifactorial diseases like congestive represent the greatest challenge faced by clinical gene therapy scientists. There is no substitute for large-scale in the advancement of the field," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA.

Explore further: Gene delivery to the lung can treat broad range of diseases within and beyond the lung

More information: William F. Penny et al, Randomized Clinical Trials of Gene Transfer for Heart Failure with Reduced Ejection Fraction, Human Gene Therapy (2017). DOI: 10.1089/hum.2016.166

Related Stories

Gene delivery to the lung can treat broad range of diseases within and beyond the lung

January 25, 2017
Targeting therapeutic genes to the lungs offers the potential to manage serious lung diseases that do not respond to other forms of treatment and to use the lungs as metabolic factories to produce therapeutic proteins for ...

Will AAV vectors have a role in future novel gene therapy approaches?

March 20, 2017
Recombinant adeno-associated virus (rAAV) vectors for delivering therapeutic genes have demonstrated their safety in multiple diseases and clinical settings over the years and are a proven and effective tool that can be used ...

Debate continues over human immune system's role in blocking therapeutic genes delivered using AAV vectors

April 5, 2017
The hypothesis that memory T cells formed in response to childhood infections may be to blame for the loss of expression of therapeutic genes delivered via viral vectors has been hotly debated, but recent clinical trials ...

Gene therapy to prevent vision loss: Studies in primates needed to optimize human trials

December 29, 2016
Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new therapies by studying ...

What causes gene transfer to trigger T cell activation and exhaustion?

April 27, 2017
Researchers are beginning to gain a clearer understanding of how the immune system responds, in both a reactive and tolerant way, to gene therapy delivered using what has become the preferred gene delivery vector, adeno-associated ...

Gene therapy for liver disease advancing with the help of adeno-associated viral vectors

December 30, 2016
Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing. The potential for continued technological improvements to expand the ...

Recommended for you

Genome analysis with near-complete privacy possible, say researchers

August 17, 2017
It is now possible to scour complete human genomes for the presence of disease-associated genes without revealing any genetic information not directly associated with the inquiry, say Stanford University researchers.

Science Says: DNA test results may not change health habits

August 17, 2017
If you learned your DNA made you more susceptible to getting a disease, wouldn't you work to stay healthy?

Genetic variants found to play key role in human immune system

August 16, 2017
It is widely recognized that people respond differently to infections. This can partially be explained by genetics, shows a new study published today in Nature Communications by an international collaboration of researchers ...

Phenotype varies for presumed pathogenic variants in KCNB1

August 16, 2017
(HealthDay)—De novo KCNB1 missense and loss-of-function variants are associated with neurodevelopmental disorders, with or without seizures, according to a study published online Aug. 14 in JAMA Neurology.

Active non-coding DNA might help pinpoint genetic risk for psychiatric disorders

August 16, 2017
Northwestern Medicine scientists have demonstrated a new method of analyzing non-coding regions of DNA in neurons, which may help to pinpoint which genetic variants are most important to the development of schizophrenia and ...

Evolved masculine and feminine behaviors can be inherited from social environment

August 15, 2017
The different ways men and women behave, passed down from generation to generation, can be inherited from our social environment - not just from genes, experts have suggested.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.