Scientists have developed a genome editing system that has successfully modified the DNA of mice with a mutation similar to that found in people with autism spectrum disorder (ASD), writes SCMP.
The article says special mice were bred for research with a mutation in the MEF2C gene, which they say is “strongly associated” with the disorder. Thus, mutations in this gene cause developmental disorders, speech problems, repetitive behavior and epilepsy, rbc.ru reports.
The developed system for editing the MEF2C gene was called AeCBE. As the study authors note, mice that received a special injection showed a decrease in behavior associated with ASD. The scientists emphasized that the potential treatment could be used not only for patients with ASD, but also for other genetic neurodevelopmental disorders.
A professor at East China Normal University told SCMP that this is the first effective treatment for mice with mutations associated with ASD. The injection would go directly into the mouse’s brain, he said, so scientists needed to learn how to safely interact with the blood-brain barrier, a group of cells that regulate the entry of foreign molecules into the brain. By studying mouse brain cells, the researchers found that AeCBE was able to “make repairs” throughout the brain with about 20% accuracy, which was enough to boost MEF2C protein levels, SCMP writes. “The treatment successfully restored MEF2C protein levels in several brain regions and reversed behavioral abnormalities in mice with the MEF2C mutation,” the paper states.
The exact cause of ASD is still unknown, but it is believed that 80–90% of cases are due to genetic predisposition. More than 100 genes have already been found that scientists associate with the occurrence of autism. But there are also environmental factors that can also contribute to the development of this disorder in a child. For example, inflammation in the mother’s body during pregnancy has been linked to an increased risk of ASD in the child. It can occur due to chronic diseases: arthritis, lupus or diabetes, and can also be triggered by obesity due to cytokines that penetrate the blood-brain barrier and attack neural networks.
Some people diagnosed with ASD as children outgrow it, achieving an “optimal outcome.” The term was coined by Deborah Fine, a professor of psychology at the University of Connecticut in Storrs. In 2013, she conducted research with 34 people diagnosed with autism. In 2016, scientists reviewed cases of “optimal outcomes” and concluded that it is possible to talk about loss of diagnosis only if it is made early. Timely, intensive behavioral intervention plays an equally important role. A large proportion of people with autism maintain symptoms consistent with the diagnosis and require therapy and support throughout their lives.
Stem cells play a crucial role in tissue regeneration and offer potential treatments for various diseases. They are present in every individual and can be administered in controlled doses to enhance their effects, aiding in the restoration of damaged nerves and regulation of the immune system. However, misconceptions and hype surrounding stem cells have led to several myths that need to be addressed.
Myth #1: Stem cell therapy is a scam since it claims to cure multiple diseases.
In reality, stem cell therapy utilizes the power of stem cells to stimulate the formation of new cells, facilitating the replacement of damaged or dead cells in the body. This versatility makes them potential treatments for autoimmune diseases like multiple sclerosis and type 1 diabetes, among others.
Myth #2: Stem cell treatment causes cancer.
While there were concerns about the risk of malignant neoplasms due to the high reproductive potential of mesenchymal stem cells (MSCs), recent tests show that this risk has not materialized. However, caution is still necessary, as MSCs’ mechanism of action can be both beneficial and potentially harmful.
The tumor growth is more typical for embryonic stem cells (obtained from embryos or in vitro fertilization materials). The use of these cells in clinical practice is limited due to ethical concerns.
However, it is important to understand that stem cell treatment is a complex medical procedure that requires the expertise of trained and experienced medical professionals. Patients should consult with their healthcare providers during all stages of the process to make sure they receive the most appropriate treatment for their specific condition.
Myth #3: Stem cells are exclusively found in embryos and obtained through abortions.
Contrary to the misconception, stem cells are not limited to embryos. Adult tissues also contain stem cells, such as those found in the umbilical cord, placenta, adipose tissue (fat), and bone marrow. The use of embryonic stem cells from aborted fetuses is highly restricted and controversial, whereas regenerative therapy primarily relies on adult stem cells, which are less contentious and more widely available.
In conclusion, stem cell research is an exciting field with significant potential for medical advancements. By dispelling these myths and understanding the actual science behind stem cells, we can make informed decisions about their applications and ensure that they are used responsibly and ethically to provide the most effective treatments for various conditions.