For kids with rare genetic disorders, customized CRISPR treatments offer hope
December 13, 2024
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(NPR) – “The for-profit sector is focusing on conditions, such as sickle cell disease, such as cancer, which are commercially viable because there are just enough people with them,” Urnov says.
The problem is, “that leaves 99.5% of folks outside of the big building that says, ‘Come here, be healed by CRISPR’ because the commercial viability is not there even though the technical feasibility is right in our hands.”
So Urnov, as well as scientists at other universities, including the University of Pennsylvania and Harvard, are trying to develop a template for groups of rare conditions that are similar enough that a gene-editing treatment for one could be easily adapted for others. (Read More)