New Ways to Cure Blood Disorders

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There have been new developments in gene-editing technology that might be able to treat sickle cell disease, and other genetic blood disorders, according to scientists. Scientists from Yale University and Carnegie Mellon University created a research team to test the gene-editing technology on mice. The experiment uses a system that was built on current gene-editing technology such as clustered regularly interspaced short palindromic repeats, or CRISPR. This approach is not like conventional methods that depend on DNA-cutting enzymes to mark specific genes. This new approach is said to have multiple positive implications for humans struggling with genetic blood disorders in the future.

“We have developed a system that uses FDA-approved nanoparticles to deliver our PNA molecule along with a donor DNA to repair a malfunctioning gene in living mice. This has not been achieved with CRISPR,” study author Danith Ly explained in a press release.

The new system uses peptide nucleic acids (PNAs) which are nano-sized synthetic molecules to open up double-stranded DNA and bind near the target site without cutting anything. In the study conducted by the team of scientists, they used this approach to target a malfunctioning gene that was associated with producing hemoglobin to treat beta thalassemia, which is a blood disorder. Not only did the scientists test this approach on mice, they also tested it on human bone marrow cells in the lab. They were able to observe a 7 percent increase in successful gene editing and higher levels of hemoglobin for several months after treatment.

“The effect may only be 7 percent, but that’s curative,” Ly added. “In the case of this particular disease model, you don’t need a lot of correction. You don’t need 100 percent to see the phenotype return to normal.”
Although the percentage might be small, the study’s authors maintain gene correction should be minimal when treating such disorders.

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