Treatment of autism spectrum disorder by regulating neurotransmitters and modifying genes

Abstract

Autism spectrum disorder (ASD) is a neurological and developmental disorder, which has many underlying causes, making it much more challenging to find an appropriate treatment to cure. It has been found that ASD is related to a wide variety of neurochemical changes, including GABA, oxytocin, and several monoamine neurotransmitters such as dopamine, norepinephrine, and serotonin. Since ASD is heterogeneous, precise technology such as PET, LP, iEEG, rs-fMRIs, and MRS had been emerged to discover the site of illness. It requires a persistent effort since the ASD phenotype is invertible instead of unchanged, which will be explained soon. Until now, only genetic modified virus (rAAV) have so far successfully incorporated with transduction and gene expression in vivo. However, whether the presence of neutralizing circulating antibodies will preclude intravenous administration is unsure. Instead of systemic delivery, intrathecal administration is discovered as a substitute, which can solve the problem and have advanced to a clinical trial. More gene therapies are hypothesized, for instance, using rAAV for gene replacement, ASOs for RNA knockdown, and CRISPR/Cas9 for gene editing. Unfortunately, these gene therapies still have a long way to go to overcome the restrictions. As a result, this research will analyze the different treatments for the ASD.