There is good news for patients suffering from the Rett syndrome as the scientists located at the University of Virginia School of Medicine is developing a gene therapy that can be of massive help for the patients who are suffering from the Rett syndrome. When a mutation occurs on the X chromosome, Rett syndrome is caused & it hugely affects the brain’s development, leading to even seizures & breathing problems. The scientist is of Indian origin & her name is Sanchita Bhatnagar & she is joining hands with another scientist at the Center for Gene Therapy to bring about relief for those battling with Rett syndrome.
Aim of Gene Therapy
The main aim of gene therapy is to cause manipulation in the cell regulators for expressing healthy copies of specific proteins called microRNAs. After finding the regulators & manipulating them to cause altercation in these regulators’ states, the systems related to this disease can be rescued to express the healthy copy of the defective gene in the cells.
This systematic usage of planned knowledge can help develop an innovative gene therapy that can slow down the disease progression & also improve movement, co-ordination & communication in patients with Rett syndrome. This approach will also help the patients in battling other genetic disorders that involve the X chromosome.
Sanchita Bhatnagar, a student of the University of Virginia School of Medicine, is pursuing a Ph.D., and she has discovered that microRNAs (tiny bits of RNA) play a primary role in the occurrence of the disease known as Rett. Rett is a sporadic genetic disorder that can disarm the children with their ability to speak, move & even breathe. She is carrying out the experimentation process to sop up the RNA bits using the microRNA’s absorbing quality.
Her excellent work in lab models has led to good quality results & thus, it has raised the hopes of the patients with such diseases to have a better quality of life. Recent experiments have been conducted on several animals who have been treated with this state-of-the-art gene therapy. The results have shown that the animals have become more mobile & they have started to move in a much faster way. The same improvements are seen in children of Rett syndrome patients can lead to a substantial positive difference.
This syndrome affects mainly 1 in 10,000 children, and generally, it is much more common in girls. The girls’ biological wiring is such that the Rett syndrome commonly affects them & this is because girls have two X chromosomes & the role of the X chromosome biology is very prominent in the occurrence of the Rett syndrome. The research has discovered the role of microRNAs in Rett as, for the first time, a microRNA has been associated with the X chromosome biology.
Bhatnagar had achieved this feat by making microRNAs sponges that can target microRNAs so that they can absorb them & then break them down. As per the statements of Bhatnagar, it was clear that they were she was not altering the genomic DNA. Still, instead, she was very clever in utilizing them as inhibitors that integrate into the gene.
Thus, with these latest developments in the field of biotechnology & genetic research, it can be stated that the cure for the Rett disorder is on its way. This can lead to patients getting some respite from its effects such as learning disabilities, developmental delays & behavioral problems.
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