Gene Therapy as a possible Candidate for Autism Treatment

Small Steps Big Dreams in Dubai, UAE plays its part in spreading awareness and knowledge to parents and caregivers for a better understanding of Autism Spectrum Disorder. With gene therapies now being considered for autism treatment, let us understand how it can prove to be a possible breakthrough for autism therapy.

Gene Therapy

Gene therapy- aka human gene transfer- is a medical field that focuses on the utilization of the therapeutic delivery of nucleic acids into a patient’s cells as a drug to treat disease. The therapy involves delivering healthy copies of a gene to replace faulty ones. This is currently also being tested for a number of brain-related disorders, including Parkinson’s and Alzheimer’s diseases.

According to Timothy Yu, a neurologist and geneticist at Boston Children’s Hospital in Massachusetts, gene therapy can help you make an actual difference and improve the quality of life for an autistic child.

Gene Therapy for Autism Treatment

In disorders defined by loss of function mutations (e.g. RS, FXS, TSC), simple gene replacement may restore synaptic function. Given the limitations imposed by imperfect gene delivery strategies, a key question is whether sufficient transduction of target cell types can be attained to exert phenotypic benefit.

According to the researches done for optimum autism treatment and autism support in Dubai, a strategy that might work is to limit gene delivery to astrocytes- non-neuronal brain cells that help regulate the brain’s chemical environment. Animal studies show that expressing the gene in just these cells alleviates symptoms of the disorder. This method can also be employed for Rhett syndrome treatment.

A review published on 27th of June in the peer-reviewed journal, Neuropharmacology, outlines the challenges of developing gene therapy for neurological disorders such as Autism Spectrum Disorder. Steven James Gray, a researcher at the Gene Therapy Center at the University of North Carolina-Chapel Hill, points out two major hurdles. The first lies in the practical difficulties of delivering the corrective copy of a gene across the large human brain. Second, the dose of the corrective copy has to be just right. Studies in animal models suggest that too much of certain genes can have harmful side-effects, and too little has no effect.

But, given the delicate dosing issues and the unpredictable nature of gene therapy, drugs being tested for these disorders are a more practical option.

The Link Between Genes and Autism

ASD is known to run in families, but the inheritance pattern is usually unknown. People with gene changes associated with ASD generally inherit an increased risk of developing the condition, rather than the condition itself. According to common autism diagnoses, we see that over 1,000 genes are thought to be linked to ASD, and many others might not be confirmed yet. Any effect they have on an individual’s risk of developing ASD is likely a mere glimpse of other potential risk factors.

Gene Therapy: CRISPR

Clustered regularly interspaced short palindromic repeat (CRISPR) is a type of gene therapy that can be used to make changes in DNA. Scientists target genetic mutations that are known to cause diseases and developmental disabilities, such as autism. So far, CRISPR technologies have been primarily studied in mice, with more recent studies taking place in monkeys.

Scientists have discovered promising applications of the technology for the effective treatment of diseases caused by specific genes. Studies have found that the impact of CRISPR on genes linked to ASD in mice and primates reduces autistic symptoms. According to their studies, they have realized the powerful potential of the gene-altering method. CRISPR can re-structure genomes to almost anything you want.

The Need for Future Studies

By way of improving autism support in Dubai, we’ve discussed various methods and strategies to work on autism. Researchers and scientists are still looking into the possibility of gene therapy as the most efficient way of treating autism. Optimum autism diagnoses can lead to opting for the best and most precise autism treatment, which can result in understanding how to tackle autism. Eventually, this could help scientists and researchers develop more targeted therapies and treatments.

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