Gene editing techniques have shown promise in early experiments that suggest they may be able to remove the extra chromosome causing Down syndrome, a condition where an individual has an extra copy of the 21st chromosome.
Down syndrome is caused by inheriting two copies of chromosome 21 from one parent and one copy from the other. Currently, there are no treatments for this condition, which can be detected in cells as early as days after fertilization.
Researchers have used the CRISPR-Cas9 gene editing system to eliminate the extra chromosome in trisomy 21 cells grown in test tubes. The technique allows it to identify the duplicated chromosome and prevent incorrect removal of one copy, resulting in a cell with only one copy from each parent.
However, this approach is not yet ready for testing in animals due to potential changes in retained chromosomes. Despite this, the researchers believe their work will lay the groundwork for more sophisticated medical interventions targeting Down syndrome.
In related news, scientists have also made progress in developing new treatments for neurological disorders using lipid nanoparticles that can bypass the blood-brain barrier. These tiny particles deliver messenger RNA instructions into brain cells, where they can help replace missing proteins or reduce harmful ones. Further research is needed to assess long-term safety and efficacy before these treatments can be tested in humans.
As one researcher noted, the findings highlight the potential of lipid nanoparticles in overcoming a major challenge in treating brain diseases – getting past the blood-brain barrier.
Source: https://www.reuters.com/business/healthcare-pharmaceuticals/health-rounds-gene-editing-may-hold-key-preventing-down-syndrome-2025-02-19