Gene therapy for the treatment of chronic heart failure **

A promising gene therapy will soon be tested in trials in the United States on persons. NanoCor Therapeutics has developed an experimental treatment called “Carfostin”: it has recently been accepted as experimental drug. The first trial stage will start later this year (2016) in patients with advanced heart failure.

Gene therapy is a technique for which genes are used to treat or to prevent diseases. Researchers are testing several gene therapy methods at the moment, including the replacement of a defect gene by a healthy one, meaning the introduction of a new gene and that the wrong gene can no longer cause any disease. This new, therapeutic gene will be inserted into the human body to help cure a disease.


Carfostin is this new, therapeutic gene. We hope this treatment will not only improve the systolic heart function, but will also be able to restore a dilated heart to its normal proportions.

Carfostin supplies the defining active form of protein phosphatase 1 Inhibitor (I-1) directly to the damaged heart cells and focuses on type 1 protein phosphatase (PP1), a serious negative regulator of calcium cycle and heart contractility. Carfostin is based on biological anode

particles (BAP) and AAV Viral Vector technology making the transfer of therapeutic genes possible, particularly in the heart muscle cells. Carfostin is an one-time treatment and will be administered through the femoral artery into the coronary ones.

Dr. R.J. Hajjar, a leading Cardiologist, co-founder of NanoCor Therapeutics and Head of a scientific Research Center at a medical faculty in New York City says:

Dr. R.J. Hajjar

“… This therapy is a feasible option for the treatment of congestive heart failure. Carfostin offers advantages compared to traditional treatment methods because it can be brought to the heart in a targeted way.

This therapy treatment is less invasive than for example a surgery…”.

Congestive heart failure

Cardiovascular disorder is globally the most important cause of death for which congestive heart failure (CHF) is the final stage. CHF is a disorder in which the heart is no longer capable of pumping a sufficient amount of blood and oxygen to the body.

CHF can be the result of a weakening of the heart muscle (stiffening) or the increase of oxygen consumption by the body tissue outside the pumping heart capacity.

Approximately 5,000,000 patients in the United States are suffering from CHF and it is one of the most common reasons why patients at the age of 65 and older are being hospitalized.


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