A new generation of a gene-editing system technology may be able to cure certain blood disorders which are genetic such as sickle cell disease. According to a new study, scientists were able to achieve this feat in mice.
An international team of researchers form of scientists Yale University and Carnegie Mellon University has had a breakthrough in their study published in the journal Nature Communications. They have successfully used a gene-editing system on mice. The experiment resulted in curing genetic blood disorders.
The innovative system that they have used builds on the current technology available for gene-editing such as the regularly interspaced short palindromic repeats, or CRISPR for short. However, the researcher’s system did not rely on DNA-cutting enzymes which target specific genes, instead, they developed a gene-editing system that allows FDA-approved nanoparticles to deliver a PNA molecule with DNA from a donor to repair genes that are malfunctioning in live mice. This result is not achievable with CRISPR technology, said the scientists in a press release.
The new and innovative gene-editing system use peptide nucleic acids (PNA) which nano-sized synthetic molecules, to unravel the double-stranded DNA so that they can bind near the target location without involving cutting methods of any kind. During their study, the researchers used this method to target and repair a gene which is associated with the production of hemoglobin. Repairing the gene led to the treatment of beta-thalassemia, a genetic blood disorder.
Although most of the research of the study was done on live mice, the scientists also tested the new gene-editing system on human bone marrow cells. Applying the same method of treatment led researchers to observe a 7 percent increase in the success of gene editing and increased levels of hemoglobin for a few months after the treatment. Although the improvement seems small, the researchers insist that gene correction should remain minimal in the treatment of these types of disorders. A large correction percentage of up to 100 isn’t in any form necessary for the phenotype of the gene to return to normal conditions.
The authors of the study are hoping that their findings will have major implications for humans in the future after more research is done on the subject.
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