Gene therapy for the brain is no longer science fiction

Voluntarist, Bitarch

Based on Konrad Lorenz’s theory of the inhibitor of intraspecific violence (Lorenz mechanism or LM), James Blair’s model of the violence inhibition mechanism (VIM), as well as a multitude of supporting evidence, including neurophysiological and genetic data, it can be confidently stated that the ability of some people to commit violence against other people is the result of a pathological impairment in the functioning of the innate violence inhibitor (LM/VIM). And since this is a pathology, it must be combated, as with any other pathology, namely through its treatment [1].

Since the impairment in the functioning of the LM/VIM is of genetic origin, treatment requires gene therapy intervention. At the neurophysiological level, specific areas of the brain are responsible for the functioning of this mechanism, which means we need to perform gene therapy on brain cells. However, the possibility of conducting such gene therapy is questioned. There is also an opinion that gene therapy cannot correct neurophysiological impairments. However, all these doubts can be easily dispelled, which I will now do.

In the past, gene therapy on brain cells was impossible because the viral vector with genetic material could not cross the blood-brain barrier—the protective filter between the circulatory and nervous systems. However, in 2003, a group of researchers found a solution to this problem, which involves using a vector small enough to be able to cross this barrier [2]. Later, a wide variety of tools for performing gene therapy on brain cells appeared, using different types of vectors and the genetic material they transport [3].

Fine, gene therapy can be performed on brain cells. But can it cure neurophysiological impairments? Let’s look at a practical example. There is a hereditary genetic disease called “Aromatic L-amino acid decarboxylase deficiency,” the symptoms of which include: mental retardation, inability to fully control the body, decreased muscle tone, seizures, and many others. It is caused by a mutation of the DDC gene, leading to a deficiency of key neurotransmitters (dopamine and serotonin).

However, quite recently (the corresponding study was published on July 12, 2021), specialists developed a gene therapy to treat this disease. A drug with a viral vector carrying the corrected version of the gene is injected directly into the patient’s midbrain, where it distributes through the tissues, and they gradually become capable of producing the missing proteins. This gene therapy has proven its safety and efficacy. Seven patients aged four to nine years, within 18 months after the injection, were freed from seizures, began trying to speak and smile, and two were even able to walk with assistance, which was considered fundamentally impossible for such a diagnosis [4].

Of course, at the moment, gene therapy is a complex and expensive technology that requires further development. However, there are absolutely no reasons to believe that the treatment of neurophysiological impairments and the restoration of nervous system functions (including the brain) is a fundamentally unsolvable task. Current research, on the contrary, proves the possibility of this. This means that the argument about the impossibility of treating LM/VIM dysfunction, based on such a claim, is incorrect.

Sources:

  1. Voluntarist (2021). Analysis of intraspecific violence as a phenomenon of human nature: norm or deviation?
  2. Ananthaswamy, A. (2003). Undercover genes slip into the brain;
  3. Ingusci, S. et al (2019). Gene Therapy Tools for Brain Diseases;
  4. Pearson, T. S. et al (2021). Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.

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