CRISPR technology report says that genetic disorder can be cured by administrating gene therapy which allows to mend mutations in the brains of foetuses in the womb.
In this treatment doctors inject a harmless virus in the feotus’s brain that infects the neurons and delivers a suite of molecules that correct the genetic faults.
Researchers are trying hard to solve this disease so they started their experimental treatments on the fetal animals and the treatment has been attempted for the first time.
The first gene therapy for adults and children were approved in last year by the US Food and Drug.
“We believe that this could provide a treatment, if not a cure, depending on when it’s injected,” said Mark Zylka, a neurologist.
Researchers suggest that the therapy will be most effective around the second trimester, when their brains are in the early stages of development.
“It had gotten to the point where I’d given up on telling people that fetal gene therapy is a good idea,” saying this Waddington added, “And now, not infrequently, people turn to me and say, ‘You know what would be a good idea? Fetal gene therapy.”
Canavan disease is an uncommon, lethal hereditary confusion in which a protein called aspartoacylase isn’t dynamic. This protein separates the amino corrosive NAA in the body.
The specialists utilized a peptide nucleic corrosive based quality altering strategy that they had recently used to fix beta thalassemia, a hereditary blood issue that outcomes in the diminished creation of hemoglobin, in grown-up mice.
Though some has risen some questions but the researchers believe that their technique might be able to achieve even higher success rates.
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