Gene-editing successfully treated Duchenne muscular dystrophy, study found - Sovereign Health Group
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04-06-16 Category: Behavioral Health

Duchenne muscular dystrophy

Variations in our genes are responsible for all of our traits and characteristics that make us unique — whether we have blond hair, blue eyes, or light or dark skin is largely dependent on our genes. Some genetic changes may also be responsible for increasing our susceptibility to certain types of mental illnesses such as schizophrenia and depression.

Gene editing: Implications for behavioral health science

One of the most important uses of gene editing technology is to treat genetic diseases (i.e., inherited medical conditions caused by abnormalities in the DNA) including fragile X syndrome, Down syndrome, sickle cell anemia, Huntington’s disease and several others. Gene editing technology has been used to either delete or insert sequences into target genes to treat genetic disorders. Scientists have recently used a gene editing tool for the first time to successfully treat a genetic disease, called Duchenne muscular dystrophy (DMD) — a devastating disease that affects about 1 of every 5,000 male births and is caused by a mutation in the dystrophin gene — inside of a fully developed living mammal.

Researchers from Duke University used clustered regularly interspaced short palindromic repeats (CRISPR) to correct genetic mutations in cultured cells from patients with DMD. Essentially, the CRISPR-Cas9 technology is used to identify a specific gene, and then the scientists can essentially “cut and paste” genetic material into parts of a gene. Another approach is to use gene therapy techniques, which involve taking CRISPR directly to the affected tissues using a non-pathogenic carrier called adeno-associated virus (AAV) to deliver the gene-editing system.

The use of gene editing tools can now be successfully implemented to treat genetic diseases, which is very exciting for the medical, psychiatric and psychological communities. There are several types of mental illnesses, including depression, bipolar disorder, attention deficit hyperactivity disorder (ADHD), autism spectrum disorders (ASDs) and schizophrenia, which have a major underlying genetic component that contributes to their development. Researchers found that these five mental illnesses can be traced back to the same inherited genetic variations in genes, which account for 17 to 28 percent of the risk for mental illness.

Environmental factors also play a role

It is important to remember that while gene editing could be useful for removing genetic susceptibility, environmental factors contributing to mental illness are still very present. In some cases, different types of mental health problems have been linked to infections, brain injury or trauma, long-term substance abuse and dependence, and other factors such as nutrition, exposure to toxins and more. For example, certain infections have been linked to brain damage and contribute to worsening the symptoms of mental illness. These infections include pediatric autoimmune neuropsychiatric disorder (PANDA) associated with the Streptococcus bacteria that is linked to obsessive compulsive disorder (OCD) and the development of other mental health conditions in children.

Epigenetic factors can further increase a person’s susceptibility to developing a number of illnesses, behaviors and other health conditions by flipping the “on-off” switch on genes compared to genetic factors that result in changes to the DNA sequence. Epigenetic factors are helpful for explaining how being exposed to certain environmental factors — including heavy metals, pesticides, viruses, bacteria, tobacco smoke, pollutants and exposure to the sun — can make us more likely to develop certain types of disease.

A huge potential

The recent introduction of gene editing technology has given rise to the possibility of changing human genes through gene therapy. Although many questions still remain regarding the ethics of changing our genes, the potential use of such technology could alter our susceptibility for disease, appearance, personality and virtually anything and everything about ourselves that is genetically based, including our genetic risk for certain mental illnesses.

Sovereign Health of California offers pharmacogenetic testing as part of our comprehensive behavioral health treatment programs for patients who have addiction, mental illness and co-occurring disorders. For more information on pharmacogenetic testing or about the treatment programs offered at Sovereign Health of California, please contact our 24/7 helpline to speak to a member of our team.

About the author

Amanda Habermann is a writer for the Sovereign Health Group. A graduate of California Lutheran University, she received her M.S. in clinical psychology with an emphasis in psychiatric rehabilitation. She brings to the team her background in research, testing and assessment, diagnosis and recovery techniques. For more information and other inquiries about this article, contact the author at

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