A New Approach in Medicine

New Gene Delivery Method Paves the Way for Advanced Brain Therapies:

On August 15, 2024 a new article was published speaking out about a new study where genetic material is being transported in the brain and for it to effectively reach cellular targets. The genes will essentially go across the blood-brain barrier and in order to do this it has to latch onto human protein and hopefully it will then distribute the genetic cargo as its called to the many, many cells in the brain. Adeno-Associated viruses are found to be the main delivering system for these therapies. These viruses are key in a sense because they start treating the disorder at their "root genetic cause." This article also goes into great detail about how this method is very effective but the blood-brain barrier can be a tricky place to be in. It's main job is to be a filter in the brain and when the gene therapies are in action, they can get filtered out too making it very difficult for it to do it's job. In one of the newer Adeno-Associated viruses, I do not believe this has been used on humans yet, but they are using mice that express the human form of one of the receptors and it looks very promising that it can be a big success in people. 

I've learned about the blood-brain barrier before in the brain, but after reading this article and finding out it can play a big role in making life saving discoveries it's very cool in my opinion. I don't know too much about these small viruses that can be used in the process, but if they start seeing success in humans then this can be huge in research and how we can treat individuals in the future. 

Sources:

https://www.nimh.nih.gov/news/science-news/2024/new-gene-delivery-method-paves-the-way-for-advanced-brain-therapies

https://www.broadinstitute.org/news/new-gene-delivery-vehicle-shows-promise-human-brain-gene-therapy

ADHD

 

This article discussed ADHD. Attention-deficit hyperactivity disorder is a behavioral disorder that develops during childhood and can be characterized by a short attention span, hyperactivity, and little to no impulse control. ADHD runs within families, the genes a person inherits from their parents play a huge role. Some with the disorder, have a hard time with school, work, and relationships with others, due to the fact that the behaviors are severe enough to interfere and cause a lack of attention. ADHD may be caused by a rare gene mutation, a mutation within a single gene is enough to trigger this disorder. Many people in my family have ADHD and it is interesting to see first hand how it may affect their day to day lives and how they do things differently than others, not being able to pay attention for long periods of time or often being forgetful. 

https://medlineplus.gov/genetics/condition/attention-deficit-hyperactivity-disorder/#resources

https://www.nature.com/articles/s41380-018-0070-0

Scientists find new genetic roots of Schizophrenia

UCLA scientists have made a major development in understanding the biology of schizophrenia. While analyzing DNA, the scientists found tons of genes and two major biological pathways that are most likely involved in the development of schizophrenia. Schizophrenia is a chronic, disabling mental illness whose symptoms can include hallucinations, delusions and cognitive problems. The illness affects about one percent of Americans. The cause of schizophrenia is not fully understood. Current medications can help reduce the symptoms, but they do not cure the disorder. The study is expected to have an impact beyond schizophrenia research because it shows a general and powerful new strategy for revealing the mechanisms of human disease.

Schizophrenia has been known to be very genetic; it often runs in families. A large genome-wide association study of individuals with schizophrenia, linked the disorder to small DNA variations at more than one hundred different locations on the human genome. Most of those locations lie outside of the actual genes, so their roles in schizophrenia have been unclear. Schizophrenia is a disorder that is known to be abnormal in the cerebral cortex. In the study, the genes linked to schizophrenia include some for brain cell receptors that are activated by the neurotransmitter acetylcholine. Variations in the functions of these receptors can help create schizophrenia.

As more studies explain the roles of these genes in schizophrenia, scientists will get a greater understanding of how the disorder develops. I am glad scientists are trying to understand the causes of schizophrenia. Through this, they should eventually be able to develop more effective treatments and be able to alleviate the symptoms from people who are suffering from it.