How safe is gene therapy?

[A. Hammouda]

Gene therapy may be the ultimate and best treatment choice for genetic diseases. One drawback that can limit its use is its high cost. But, is it safe?

[A. Hammouda]

By gene therapy, a new functional gene is introduced into the body cells. It produces the protein that was supposed to be produced by an innate gene. Due to the fact that the innate gene is missing, non-functional, or malfunctional, gene therapy becomes a must.

As with any medical procedure, some complications may arise. The body may react adversely to the newly produced protein, considering it a foreign protein. This may lead to failure of gene therapy, with immune reaction of the body against the produced protein.

Complications may arise from the gene-delivering vector. A common type of vector used is adeno-associated virus (AAV). This vector is used to carry the required gene to the target cells. Although the manufacturers ensure that the virus used is infective but not pathogenic, complications still are encountered.

Guidelines should be followed before, during, and after gene therapy. However, this does not guarantee a zero per cent complications. Recently, we have had a shocking news of two fatalities resulting from gene therapy of spinal muscular atrophy:

Death after Zolgensma – Two Cases Acknowledged – Bioinformatics Hub

This $2.1 million gene therapy is the promising life-saving one-time gene therapy for patients with SMA. Thousands of patients have been successfully treated according to the manufacturer, Novartis

The bottom line is: Medicine is not Mathematics.

• This reply was modified 2 years, 3 months ago by BioHub.

[Fazil Hussein]

One important complication of gene therapy is cancer.

 

[A. Hammouda]

That is right, especially when using lentivirus for delivering the gene of interest to the host cell. Lentivirus is a retrovirus (RNA that is integrated as its complementary DNA in the host genome). Random insertion of the virus in the host genome may cause insertion mutagenesis. An oncogene may be activated and cause cancer. Early trials for treatment of boys with X-linked severe combined immune deficiency showed the development of some leukemias (blood cancers). Therefore, stringent safety studies should be conducted for new gene therapies, and the patients should be monitored for years after the treatment.

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