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Advantages and Disadvantages of Somatic Gene Therapy

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Advantages and Disadvantages of Somatic Gene Therapy

Somatic gene therapy is a type of genetic medicine that seeks to cure diseases only in the affected individual, not their descendants. It offers an alternative to standard medical therapies like blood transfusions or organ transplantation and has been found to be safer than other genetic treatments.

Somatic gene therapy offers several advantages over other methods for gene editing, including targeting disease-causing DNA in nonreproductive cells – those which don’t produce eggs or sperm. By restricting editing to these non-reproductive cells, there’s less chance that unintended changes related to editing will be passed down through generations.

Also, somatic gene therapy helps reduce the risk of introducing other genes not intended to be corrected that could create new issues in patients. While other forms of gene therapy pose this same concern, it’s less relevant with somatic gene therapy since only edited DNA is delivered directly to cells within the body.

Other advantages of somatic gene therapy include its versatility in treating various illnesses and its potential to be curative for those with genetic diseases. It has particularly proven successful at curing inherited conditions like cystic fibrosis and hereditary cancers such as leukemia.

Another advantage of somatic gene therapy is its affordability; it only requires a few injections into patients’ bodies and there’s no long-term commitment required; thus, patients don’t need to continue taking it throughout their lives.

Somatic gene therapy can be used to treat hereditary disorders that involve one gene or several genes, such as heart disease, high blood pressure, Alzheimer’s disease and arthritis. It may also be utilized to correct errors in a single gene like thalassaemia.

Unfortunately, somatic gene therapy cannot be used to treat hereditary cancers that involve multiple genes. When multiple mutations are involved, it becomes harder to correct all the abnormalities causing a condition.

A potential drawback of somatic gene therapy is its potential to introduce toxicity and immune or inflammatory responses into the body. This is because viral vectors used for delivery may get reprogrammed by cells within the body and regain their capacity for disease.

Some people worry that the potential toxicity and side effects of somatic gene therapy may outweigh any benefits it might bring to some individuals. However, gene therapy has only been around for a few years, so we have yet to fully assess its long-term consequences.

NIH is striving to address these obstacles by developing genome editing tools that are safe and reliable in humans, making them widely accessible to researchers. Through the Somatic Cell Genome Editing program of the NIH Common Fund, these resources will be made available so researchers can quickly translate gene and cell therapies into clinics for as many patients with genetic diseases as possible.

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