Gene Editing Technique Promises To Treat Thalassemia In Womb

Gene Editing Technique Promises To Treat Thalassemia In Womb

Gene Editing Technique Promises To Treat Thalassemia In Womb(Representational Image)

New Delhi: Scientists have achieved a new breakthrough in medical science, discovering the potential of a gene editing technique to treat beta thalasemmia, a geneic blood disorder, paving the way for treatment of various genetic conditions during early stages of foetal development.

In the blood disorder condition, the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body, gets lowered.

Carnegie Mellon University in the US conducted this research by using a peptide nucleic acid (PNA)-based gene editing technique to successfully treat beta thalassemia in adult mice.

PNAs are synthetic molecules that combine a synthetic protein backbone with the nucleobases found in DNA and RNA.

According to the researchers, the mice that were treated using PNA while in utero, showed normal levels of haemoglobin, less spleen enlargement and increased survival rates.

Danith Ly, professor from the varsity," Early in embryonic development, there are a lot of stem cells dividing at a rapid pace. If we can go in and correct a genetic mutation early on, we could dramatically reduce the impact the mutation has on foetal development or even cure the condition."

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The study published in the journal Nature Communications, showed that just one injection of the PNA complex into the amniotic fluid during gestation led to dramatic improvements in the mice's symptoms and correction in 6 per cent of the beta thalassemia causing mutations.

No off-target effects of the technique from the treatment were discovered, suggesting that the method would be better than other gene editing techniques like CRISPR/Cas9, which can erroneously damage off-target DNA.

Ly said," The PNA technique is more ideal for therapeutics. It doesn't cut the DNA, it just binds to it and repairs things that seem unusual. We looked at 50 million samples and couldn't find one off-site error when we used our PNA gene editing technique."

As per the reports of World Health Organisation, nearly 1.1 per cent of couples worldwide are at risk for having children with a haemoglobin disorder and 2.7 per 1,000 conceptions are affected.

Largest number of patients with thalassemia major in the world are reprted in India, which is about 1 to 1.5 lakh, around four per cent of the population are carriers of the beta thalassemia trait.

The technique might help in achieving even higher success rates if they can administer it multiple times during gestation, the researchers said.

(Inputs From News Agency IANS)

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