In Feb. 2025, researchers from Mie University in Japan developed a method for removing the third copy of chromosome 21, the structure responsible for causing Down syndrome, through the gene editing tool [CRISPR]. CRISPR allows scientists to pinpoint targets in DNA that may contain a mutation and remove them using an enzyme called Cas-9.
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Down syndrome is the most common chromosomal form of intellectual and physical disability, resulting in large deficits in cognitive and motor development. If CRISPR can be applied in clinical use to treat Down syndrome, it could impact one in 691 people born.
The currently approved gene therapies work “ex vivo”, meaning they are given to blood cells outside of a person’s body that then circulate throughout their system. …
However, the proposed therapy for Down syndrome takes place at the embryonic level, which means parents have to go through IVF for the procedure to take place.
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“If you can correct one thing, you can correct everything. And the question is always where you draw the line,” [says Dr. Stephanie Wong-Noonan, a professor of biology at Carnegie Mellon.] “What things will we correct? Or should we correct?”
