Gene Therapy for Autism: Altering Gene Sequence for Treatment

Gene therapy usually means that researchers are looking at gene insertion or gene-editing methods to treat or prevent conditions by changing the DNA sequence in one’s genes. 

Gene Therapy for autism is a new and emerging area of gene therapy research. Autism, which cannot be cured but can be effectively managed with behavioral therapies, is currently diagnosed based on verbal communication, nonverbal communication, and social interactions.

Evaluating gene therapy for autism would mean looking at gene editing to promote better speech or encourage more social behavior. It is essential to note that gene therapy for autism is an emerging field, meaning there is not so much work on gene therapy for autism conducted so far.

A 2013 study published by the Journal of Neuroscience showed gene therapy could help improve social behavior in a mouse model for autism. In this study, researchers injected gene therapy into mice with three different genes associated with autism.


How Does Gene Therapy Work?

The behaviors associated with ASD include deficits in social interactions and verbal and nonverbal communication and restricted or repetitive activities. The cause of ASD is not yet fully understood, but it is likely a combination of genetics and environmental factors.

The exact cause or causes of ASD are unknown, but several factors likely work together to produce the behaviors associated with autism. Researchers use the term “complex genetic trait” to describe this set of symptoms because several genes are likely involved, as well as environmental factors that may trigger the symptoms.

Gene therapies were used to the cerebellum and the striatum – two brain regions linked to autism. When they later put these mice into an environment with other mice, they showed less anxiety and more social behavior than the controls. 

Gene Therapy for autism could also be used as a form of gene therapy for Fragile X syndrome or Rett syndrome, both of which have been linked to autism.

Autism spectrum disorder (ASD) is a neurodevelopmental disorder with a complex genetic etiology-set of causes of the disease.

The genetic basis of ASD is complex. For example, no single gene is responsible for the disorder. It appears that mutations, errors of development, may play a role in some cases. Some of these mutations may be inherited, while others may appear spontaneously. These new or de novo mutations occur in genes that regulate brain growth and development, synaptic function, and neuronal signaling.

ASD is differentiated from other developmental disorders by a distinctive pattern of symptoms and by the timing of when they begin. ASD begins during infancy or early childhood, with difficulties in social interaction as the most distinctive symptom. 

People with ASD often have difficulty seeing, hearing, and relating to others either verbally or non-verbally. It is now known that the symptoms of ASD begin early in childhood and tend to persist throughout a person’s life.

We at Small Steps provide every piece of information and experts consultation to ASD patients and their families.

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